Abstract

Huntington’s disease (HD) is an inherited brain disorder caused by a flawed gene sequence. This gene produces a noxious protein responsible for the gradual death of nerve cells in an area of the brain called the striatum. When these brain cells die off, patients may experience involuntary jolted movement (chorea), loss of stability, retention difficulties and severe temper alterations. As present medications only regulate these symptoms without actually stopping the underlying brain damage, stem cell therapies have developed into a vital focus of current research, with primary objectives to protect dying tissue and restore lost brain functions. The various stem cell approaches that are currently being used and in further development for Huntington’s disease include: embryonic stem cells (ESCs); induced pluripotent stem cells (iPSCs); neural stem cells (NSCs); and mesenchymal stem cells (MSCs). 

ESCs, NSCs and patient-derived iPSCs hold the powerful capability to progress into working brain neurons to replace dead cells. Furthermore, iPSCs possess the ability to be transformed into disease phenotype models, allowing scientists to experiment with new drugs and explore the progression of HD. MSCs work mainly as protective biofactors that release essential growth proteins, specifically brain-derived neurotrophic factors (BDNFs), and reduce brain inflammation to keep any existing neurons alive. 

Pre-clinical rodent trials have proven successful in reflecting improvements in balance, movement and brain health following the implementation of stem cell-based treatments. However, there are still many obstacles current researchers face, including maintaining the life of transplanted cells over time, stopping the body from rejecting treatment, preventing abnormal cell growth and placing cells safely deep inside the brain. Overall, stem cell treatment is actively revolutionising how scientists understand and treat Huntington’s disease.

Introduction

Huntington’s disease (HD) is an inherited autosomal dominant neurodegenerative disorder, caused by a CAG expansion within the huntingtin gene (An et al., 2012; Saudou & Humbert, 2016). The huntingtin protein (HTT) is comprised of 3,144 amino acids, with the CAG repeats providing essential genetic instructions for a polymorphic polyglutamine (polyQ) stretch. While all humans contain CAG repeats (ranging from 9-35 repeats with a median of 17-20), patients presenting with Huntington’s disease contain an abnormal number of repeats (>35) (Kremer et al., 1994). This mutation causes more huntingtin protein to be made and thus harms the neurons in the brain, particularly in the striatum, a part of the brain that controls movement, coordination and behaviour. Therefore, patients with HD may experience involuntary movements, worsening cognitive abilities and psychiatric problems (Shah, Mansour & Lucke-Wold, 2025). The average age of onset for the disease is 35 years; however, patients may present with symptoms earlier or later. Furthermore, the age of onset has proven to be directly correlated with the quantity of CAG repeats, with a larger sum of repeats resulting in earlier onset (Csobonyeiova, Polak & Danisovic, 2020). Huntington’s disease is fatal, with an average continuation of a patient’s lifespan of 15-20 years post-onset (Schulte & Littleton, 2011). 

Currently, medications and therapies are being developed to further aid patients with HD; however, there are none that can replace the neurons and thus are not able to cure the disease. Nevertheless, such medications prove helpful in the minimisation of HD symptoms. Stem cell therapies have been recognised as a potential treatment for neurodegenerative diseases, including Huntington’s disease. Neural stem cells (NSCs), mesenchymal stem cells (MSCs), embryonic stem cells (ESCs) and induced pluripotent stem cells (iPSCs) are currently being tested to further treat HD symptoms, with scientists further assessing the capabilities of stem cells to slow the progression of the disease. However, there are many safety measures that must be taken into account before any product or therapy can be publicly used (Kerkis et al., 2015).

This paper outlines different stem cell therapies for HD patients, while considering the risks and limitations that arise for each treatment, namely in ensuring both the success of transplantation and ethical methodology when harvesting stem cells. Furthermore, the paper recognises the role of brain-derived neurotrophic factors (BDNFs) alongside stem cell therapies. As members of the neurotrophin family of growth factors, BDNFs are responsible for ensuring the survival and appropriate function of striatal neurons; hence, a correlation between the depletion of BDNF and Huntington’s disease was established, accounting for many of the side effects of HD that impact cognitive, behavioural and motor function (Azman & Zakaria, 2025).

Methodology

This comprehensive review was conducted using a standardised and structured approach to evaluate diverse stem cell therapies and complementary therapeutic interventions for Huntington’s disease. Primary information was gathered through the PubMed database using specific searches, such as “iPSCs Huntington’s disease“, “NSCs Huntington’s disease” and “stem cells Huntington’s disease“, and precise language focused on the aforementioned stem cells. Further research was conducted via PubMed and Google Scholar to investigate alternative (non-stem-cell-based) therapies for the treatment of Huntington’s disease. These searches identified relevant peer-reviewed journal articles, preclinical animal rodent trials and developing clinical safety evaluations. Prior to drafting, a detailed outline was planned to arrange the paper, and all the sources were cited instantly to build an accurate bibliography. 

The information was both scrutinised and synthesised with the reader in mind, ensuring that any complex facts and medical terms, such as the various stem cell therapies and their role, were clearly defined and easy to follow. Furthermore, to analyse the main types of stem cell therapies, this comprehensive review includes and credits existing, non-stem cell treatments to provide an unbiased and complete scope of the therapies currently implemented in the treatment of HD.

Neural Stem Cells

Neural stem cells are multipotent and can differentiate into the three major types of the central nervous system: astrocytes, neurons and oligodendrocytes, making them a promising frontier in modern medicine (Shah, Mansour & Lucke-Wold, 2025). Researchers are exploring the potential of transplanted NSCs to actively restore and support injured neural structures as opposed to exclusively symptomatic or pharmacological management of central nervous system damage (Capitini et al., 2023). These multipotent progenitors have the ability to integrate with pre-existing circuitry, replace lost cellular populations or regain any cellular population, and exert critical paracrine effects after transplantation. In particular, transplanted NSCs secrete crucial neurotrophic factors, predominantly brain-derived neurotrophic factor, that benefit or enhance endogenous cell survival, promote synaptic plasticity and reduce cellular stress in damaged brain tissue (Capitini et al., 2023; Shah, Mansour & Lucke-Wold, 2025). 

The potential of NSCs has been shown mainly in preclinical studies where researchers investigated whether transplanted cells can survive in the brain and reduce local neuronal damage. Transplanted human NSCs have demonstrated considerable biological efficacy in neurodegenerative rodent models, including rat models and transgenic mouse lines (Capitini et al., 2023). Neural stem cells have proven successful in differentiating into different functional neuronal subtypes, sending out axons, establishing synaptic connections with host networks and increasing the levels of protective trophic factors (Shah et al., 2025). In addition, NSC grafts have been frequently shown in preliminary studies to reduce the accumulation of harmful intercellular proteins and induce significant, long-term improvements in behaviour, cognition and motor function compared to sham-treated controls (Capitini et al., 2023; Shah et al., 2025). 

The transition from animal models to human clinical studies and application represents the most critical hurdle in stem cell research and medicine. Recently, researchers marked a major milestone with the launch of REGEN4HD, a landmark Phase 1b/2a clinical trial testing a pluripotent stem cell-derived NSC therapy in human subjects (International Society for Stem Cell Research [ISSCR], 2024; Precision Medicine Online, 2024). With a test group comprised of early-stage symptomatic adult patients, the trial was designed primarily to answer fundamental safety questions regarding surgical delivery, cell tolerability and graft stability rather than proving definitive curative efficacy, meaning that the trial was not looking for the guarantee of a cure but rather focused on collecting more data for further research (ISSCR, 2024). Furthermore, the trial focused on identifying the maximum tolerated dose while collecting exploratory biomarker and imaging data to guide the design of future late-phase clinical studies (Precision Medicine Online, 2024). 

While NSC developments are promising, many biological, technical and ethical constraints remain. Traditionally, research has been heavily reliant on foetal-derived NSCs, which result in significant ethical controversies and severe limitations in supply, isolation and scalability (Capitini et al., 2023). The use of alternative human pluripotent stem cells, which include both embryonic and induced pluripotent stem cells, as the basis for modern clinical platforms is a key technological turning point that allows for the standardised manufacturing of products; in particular, iPSCs present a solution that does not require the use of foetal tissue (ISSCR, 2024; Precision Medicine Online, 2024). 

However, there are still wider scientific challenges for all NSC applications. A major challenge of host immune rejection often necessitates systemic or localised immunosuppressive therapies for graft recipients that pose their own health risks (Shah et al., 2025). In addition, technical issues persist in the development of accurate cell differentiation, long-term survival and functional integration within complex human neural networks (Capitini et al., 2023). Safety investigations must also carefully evaluate or test tumorigenic potential; while lineage-committed NSCs, which are the unspecialised cells’ permanent choice of turning into a specific type of mature cell, generally exhibit a relatively lower risk of tumour or teratoma formation compared to undifferentiated pluripotent stem cells, the risk is reduced rather than completely eliminated (Shah et al., 2025). The unspecialised stem cells in a graft may multiply and proliferate quickly and abnormally after transplantation. 

Overall, NSCs provide strong mechanistic rationale, but their therapeutic benefit in humans remains to be demonstrated following the establishment of ongoing clinical safety trials. 

Mesenchymal Stem Cells

Mesenchymal stem cells have emerged as a significant area of clinical investigation for treating various brain disorders, as they are easy to acquire, relatively safe and release beneficial healing chemical compounds (Rossignol et al., 2014). These adult stem cells can be obtained from bone marrow, body fat, umbilical cord tissue or cord blood (Olson et al., 2012). As these specified stem cell treatments come from adult tissue rather than embryos, foetuses or adolescents, MSCs tend to avoid the major ethical controversies that are linked to other stem cell treatments (Olson et al., 2012). The main goal of implementing MSCs in the treatment of Huntington’s disease is to preserve vulnerable neurons from degeneration and decrease tissue inflammation (Dey et al., 2010; Snyder et al., 2010). 

MSCs address the underlying pathology of HD by directly resisting the toxic cellular environment that is created by the abnormal huntingtin (mHTT) protein accumulation (Rossignol et al., 2014). By secreting bioactive molecules, MSCs alleviate contained mitochondrial dysfunction, decrease persistent neuroinflammation and provide crucial trophic support to damaged striatal pathways (Olson et al., 2012). As a result of untreated striatal cell loss being both permanent and irreversible, the primary objective for stem cell intervention is to preserve vulnerable host neurons before motor and cognitive impairment becomes lethal (Snyder et al., 2010). 

The therapeutic mechanism of MSCs is significantly dependent on their ability to secrete essential neurotrophic and anti-inflammatory factors. Transplanted MSCs release substantial concentrations of brain-derived neurotrophic factors, glial cell line-derived neurotrophic factors (GDNFs) and vascular endothelial growth factors (VEGFs), which directly counter the local BDNF deficit attributes of HD pathology (Dey et al., 2010). Additionally, MSCs wield strong immunomodulatory effects by discharging anti-inflammatory cytokines, reducing microglial activation and weakening chronic neuroinflammation in the degenerating striatal microenvironment (Rossignol et al., 2014). Research scientists have also used genetically modified MSCs that are designed to over-produce BDNFs to demonstrate that targeted nutrient distribution enhances endogenous neuronal survival and slows down structural brain weakening (Olson et al., 2012). 

Preclinical studies in transgenic HD (TgHD) rodent models, including R6/2 and YAC128 mouse models, have yielded very promising outcomes (Dey et al., 2010; Snyder et al., 2010). Rodents given the MSC transplant have shown reduced brain contraction, less buildup of toxic abnormal proteins and noticeable refinements in movement, stability and running experiments juxtaposed with control groups (Dey et al., 2010; Rossignol et al., 2014). Specifically, in YAC128 mice, injecting MSCs that were altered to release a surplus of BDNF allowed the mice to stay on the rotating balance rod considerably longer and decreased striatal brain loss (Dey et al., 2010). Additionally, in rodent trials, human-derived MSC injections reduced striatal tissue loss and stimulated natural repair cells for up to 30 days in HD mice (Snyder et al., 2010). Early MSC safety trials in humans have shown that transporting MSCs immediately and directly into the central nervous system is generally harmless, and the immune system does not typically reject them; however, further experimentation must be conducted within the field of MSC technology before definitive conclusions of safety can be determined (Olson et al., 2012). 

Despite these promising outcomes, major obstacles still remain. Unlike pluripotent stem cells, MSCs cannot turn into actual, functional brain neurons that connect into the patient’s present brain pathways (Snyder et al., 2010). Consequently, the benefits of MSCs are limited to a shorter duration than alternative stem cell treatments (Rossignol et al., 2014). Moreover, transplanted MSCs are prone to dying off within a few weeks or months within the body, which may require frequent surgeries to keep nourishing the brain with protective proteins (Dey et al., 2010). Additionally, variations in the quality of stem cells (dependent on the donor’s age and quality of life) complicate the process of establishing a standardised dosage/treatment (Olson et al., 2012). While MSCs show promising potential for slowing down symptoms and improving the patient’s quality of life, researchers must continue to assess the side effects of treatments and conduct more experimental human trials before MSC transplant can become standardised.

Embryonic Stem Cells

Embryonic stem cell therapy could significantly impact the treatment given for Huntington’s disease by replacing brain cells, delivering protective factors such as BDNF and potentially providing the opportunity to slow the progression of the disease. However, this therapy remains in an experimental stage, and thus not all aspects of this treatment are yet known. While ESCs could further aid HD, there are multiple factors to take into account, such as safety, ethical and practical rules (Shah, Mansour & Lucke-Wold, 2025). 

In Huntington’s disease, the brain loses medium spiny neurons (MSNs) – a type of nerve cell – in the striatum, causing certain necessary proteins in the body which protect neurons, such as BDNF, to become reduced (Shah, Mansour & Lucke-Wold, 2025). Embryonic stem cells are pluripotent, meaning they are “capable of developing into differentiated cells” (Biology Online, 2021). Consequently, scientists are able to convert embryonic stem cells into the specific type of neuron necessary to help in the treatment of HD; however, in order for these to have an effect, they must first be transplanted into the patient’s brain. Following successful transplantation, they may either replace the dead neurons, boost proteins within the brain (BDNF) or slow the progression of HD. In addition, ESCs can also self-renew, meaning that once inserted into the body, they divide and produce more stem cells (Chuang, 2015). 

To achieve a successful replacement of damaged neurons, a derived ESC cell must be integrated into the brain circuit. If these transplanted neurons respond well to the existing brain network, they can restore movement and cognition for the patient, as well as communication. This is especially useful for patients who have already developed HD symptoms (Nih.gov, 2026). 

Furthermore, the ESC therapy can be used to boost proteins that support the growth, protection and survival of neurons, in order to slow the progression of neuron death. Elevated proteins are achieved by programming derived ESCs to produce BDNF, protecting the patient’s initial neurons as well as those later transplanted. Consequently, ESCs can improve a patient’s mobility performance and support the development of their neurons. However, when tested on animals, these effects were not consistent; thus, more testing still needs to be conducted before an accurate conclusion can be made (Zimmermann et al., 2016). 

Further limitations of ESC therapy include the difficulties of removing all faulty HD genes from the cell within each patient’s body. As Huntington’s disease is caused by an inherited mutation, the disease could keep damaging the patient’s original neurons and thus could affect the transplanted neurons as well. Therefore, cell replacement may be more effective if performed alongside other therapies, such as gene editing, to limit the harmful huntingtin protein (Shah, Mansour & Lucke-Wold, 2025). 

Similarly, there are many safety concerns that should be taken into account before commercialised ESC treatment. If the ESCs were to remain immature following implantation, they could grow uncontrollably and later form a tumour. It is therefore essential that medical specialists develop a reliable method to create the proper neuron type. Moreover, the transplant is further complicated as the neurons would need to be inserted correctly into the striatum through brain surgery, presenting substantial risks. As well as this, the immune system could reject the transplanted neurons, resulting in the consequent administration of drugs and pharmaceuticals to protect the immune system (Shah, Mansour & Lucke-Wold, 2025). Ethical concerns are also present within this solution, as the human ESCs are created from an early embryo and thus this usually involves having to destroy the embryo.

Induced Pluripotent Stem Cells

Pluripotent stem cells (PSCs) represent a group of cells that can be differentiated to perform a specialised role within the body. The two primary groups of PSCs include embryonic stem cells, recovered from an embryo prior to specialisation and induced pluripotent stem cells (iPSCs). iPSCs are stem cells that have originated from any mature, adult cell within the body before being reprogrammed to retain a pluripotent state, acquiring the capability to differentiate across all three germ layers and continuously “self-renew” (Wang et al., 2026). 

iPSCs serve in the treatment of HD by modelling the disease progression, trialling pharmacological therapies and acting as a cell replacement for damaged neural pathways. iPSCs are particularly promising in regard to their method of harvesting, which bypasses the primary ethical concerns associated with alternative PSC treatments such as ESCs (Wang et al., 2026). Additionally, as iPSCs can be generated from both healthy donors and HD patients, treatments have the potential to be specifically tailored to an individual presenting with HD. 

The metabolic impact of HD was extensively assessed in trials conducted by An et al. (2012) and Rysankova et al. (2026) through the use of HD-iPSC models and transplantations into a HD mouse and HD minipig, respectively. An et al. (2012) evaluated the potential for patient-derived HD iPSCs to be genetically corrected by replacing the expanded CAG repeat (via homologous recombination), successfully correcting two iPSC clones (C116 and C127) so as to reduce the length of the huntingtin protein. The corrected iPSCs indicated a reversal of abnormalities present in the onset of HD (including increased cell death and impaired mitochondrial function) and furthermore, survived in the striatum following transplantation into a HD mouse model. Pre-implantation, both BDNF levels and mitochondrial respiration were restored to a standard level, and corrected cells were capable of differentiating into striatal neurons (DARPP-32- and GABA-expressing neurons). However, the initial targeting efficiency to correct the iPSC clones was low, with only 1% of the total colonies correctly targeted. Rysankova et al. (2026) investigated early metabolic and genetic changes caused by the mHTT gene in transgenic Huntington’s (TgHD) minipig iPSCs. Fibroblasts (connective tissue cells that serve as a starting material for iPSCs) from both wild and TgHD minipigs were reprogrammed into iPSCs, successfully expressing pluripotency (indicated by biomarkers OCT4, SOX2, SSEA4 and NANOG) and differentiating into all three germ layers. Significantly, increased VEGF expression, HK2 and GAPDH in the TgHD iPSCs suggested that abnormal metabolic function may occur at the PSC stage, prior to differentiation into mature neurons, revealing the progression of HD before obvious neurodegeneration develops. 

Furthermore, researchers are capable of transforming pluripotent stem cells into three-dimensional, lab-grown masses of brain tissue (brain organoids) to effectively model cortical development and reveal particular disease phenotypes. While organoids present greater therapeutic potential than other 2D models, their structural complexity results in both a costly and technical growth period. In particular, the methodology for neural organoid formation requires a constant medium supply, complemented by spinning bioreactors, spanning over the course of months and resulting in a large sum of on-costs. However, efforts to minimise limitations for brain organoid development have been made by Tachibana (2018), with the implementation of “organoids-on-chip”, which have proven successful in producing more suitable and reproducible culture systems as a result of introducing biosensors and microfluidic channels. If the development of 3D brain organoids were to prove successful and sustainable, researchers would have the opportunity to transform iPSCs in order to map HD progression and, most significantly, analyse the impact of treatments on larger neural networks as opposed to isolated cell colonies. 

Csobonyeiova, Polak and Danisovic (2020) outline that in order for future success in HD treatments, iPSC-derived medium spiny neurons must undergo genetic correction prior to transplantation, recognising the various approaches to gene silencing/editing including RNAi, shRNA, antisense oligonucleotides (ASOs) and clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9. Notably, Csobonyeiova, Polak and Danisovic (2020) retain that CRISPR/Cas9 represents the most effective option, permanently eliminating targeted genes and inhibiting mHTT expression. Yet, CRISPR/Cas9 may pose limitations in ensuring the safety of the technology, as the molecular alteration of DNA has the capacity to result in unintended genetic modification and the disruption of healthy genes (Davies, 2019).

Alternative Treatments

While emerging stem cell treatments have the potential to significantly improve the quality of life for many patients with Huntington’s disease, other treatments have similarly demonstrated their potential in managing the progression of HD. Huntington’s disease has no cure; thus, doctors have had to explore alternative methods of helping patients. Current treatments primarily target the physical and psychiatric symptoms, with conventional therapies relying on a combination of FDA-approved targeted drugs, general psychiatric medications and supportive therapies (Johns Hopkins Medicine, n.d). Currently, dopamine depleters are also used, namely tetrabenazine (Xenazine) and deutetrabenazine, which are specifically approved to treat chorea (involuntary movements) by reducing the amount of dopamine available in the brain. In addition, antipsychotics may be implemented to help with hallucinations and behavioural outbursts as well as to treat chorea (Johns Hopkins Medicine, n.d). Finally, psychiatric and mood medications, including antidepressants and mood stabilisers, have proven effective in managing the emotional and behavioural changes that often arise as a result of neurological decline (Salman et al., 2025). 

Discussion

Ultimately, the implementation of stem cells (notably NSCs, MSCs, ESCs and iPSCs) to both treat and model Huntington’s disease remains a promising pathway for the future prospects of the disease. Currently, NSCs, MSCs and ESCs are being investigated for their protective qualities for neural cells, while NSCs, ESCs and iPSCs have been identified as holding the capacity to differentiate into neural cells and actively replace damaged neurons within the brain. Furthermore, MSCs and NSCs have been recognised for their anti-inflammatory properties, while iPSCs remain the primary cell used in modelling the progression of HD by means of two-dimensional cell samples and three-dimensional brain organoids. 

However, the eventual success of stem cell treatments is dependent on continuous scientific trialling and experimentation, with a particular focus on their impact on human physiology. While animal models (namely rodents and minipig trials) have provided a strong framework for the establishment of stem cells as a viable treatment for HD, continued assessment of the impact of stem cells on human physiology can be achieved through both further experimentation with human-derived HD models and continued human trials. 

Likewise, the ethical harvesting of stem cells must remain a driving factor in the exploration of stem cell therapies. Consequently, the future for stem cell treatments in HD may neglect embryonic harvesting, so as to ensure the safety of the donor and maintenance of public trust. Furthermore, the collection and/or differentiation of stem cells must be achievable on a larger scale in order for stem cell therapies to become a commercialised treatment. 

Therefore, the future of stem cell-based approaches in treating Huntington’s disease is not only dependent on how effectively stem cells can target the presence, symptoms and progression of the disease, but also on the ability for treatment to be replicated across broad patient profiles and produce reliable results. 

An, M.C., Zhang, N., Scott, G., Montoro, D., Wittkop, T., Mooney, S., Melov, S. and Ellerby, L.M. (2012) Genetic Correction of Huntington’s Disease Phenotypes in Induced Pluripotent Stem Cells. Cell Stem Cell, 11(2), pp.253–263.

Biology Online (2021) Pluripotent – Definition and Examples – Biology Online Dictionary. Biology Articles, Tutorials & Dictionary Online [online]. <https://www.biologyonline.com/dictionary/pluripotent>

Capitini, C., Garcia, M., Rossi, P. and Smith, A. (2023) Neural stem cell transplantation in neurodegenerative diseases: Preclinical evidence and translational perspectives. Brain Research, 1812, 148390.

Chuang, J.-H. (2015) Neural differentiation from embryonic stem cells in vitro: An overview of the signaling pathways. World Journal of Stem Cells, 7(2), 437.

Conner, L., Srinageshwar, B., Bakke, J., Dunbar, G. and Rossignol, J. (2023) Advances in stem cell and other therapies for Huntington’s disease: An update. Brain Research Bulletin, 199, 110673.

Csobonyeiova, M., Polak, S. and Danisovic, L. (2020) Recent Overview of the Use of iPSCs Huntington’s Disease Modeling and Therapy. International Journal of Molecular Sciences, 21(6), 2239.

Davies, B. (2019). The technical risks of human gene editing. Human Reproduction, 34(11).

Dey, N.D., Bombard, M.C., Roland, B.P., Davidson, S., Lu, M., Rossignol, J., Sandstrom, M.I., Skeel, R.L., Lescaudron, L. and Dunbar, G.L. (2010) Genetically-engineered mesenchymal stem cells reduce behavioral deficits in the YAC128 mouse model of Huntington’s disease. Behavioural Brain Research, 214(2), pp.193–201.

International Society for Stem Cell Research (2024) First clinical trial of pluripotent stem cell-derived neural stem cell therapy. ISSCR News [online]. <https://www.isscr.org/isscr-news/first-clinical-trial-of-pluripotent-stem-cell>

Jeon, I., Lee, N., Li, J.-Y., Park, I.-H., Park, K.S., Moon, J., Shim, S.H., Choi, C., Chang, Da J., Kwon, J., Oh, S.-H., Shin, D.A., Kim, H.S., Do, J.T., Lee, D.R., Kim, M., Kang, K.-S., Daley, G.Q., Brundin, P. and Song, J. (2012) Neuronal properties, in vivo effects, and pathology of a Huntington’s disease patient-derived induced pluripotent stem cells. Stem Cells, 30(9), pp.2054–2062.

Kerkis, I., Haddad, M.S., Valverde, C.W. and Glosman, S. (2015) Neural and mesenchymal stem cells in animal models of Huntington’s disease: past experiences and future challenges. Stem Cell Research & Therapy, 6(1).

Kremer, B., Goldberg, P., Andrew, S.E., Theilmann, J., Telenius, H., Zeisler, J., Squitieri, F., Lin, B., Bassett, A., Almqvist, E., Bird, T.D. and Hayden, M.R. (1994) A Worldwide Study of the Huntington’s Disease Mutation: The Sensitivity and Specificity of Measuring CAG Repeats. New England Journal of Medicine, 330(20), pp.1401–1406.

McCaughey-Chapman, A. and Connor, B. (2026) Huntington’s disease human lateral ganglionic eminence precursors differentiate into functionally mature medium spiny neurons exhibiting pathology. Stem Cells, 44(8).

Neuroscience News (2026) World-First Embryonic Stem Cell Trial for Huntington’s Opens. Neuroscience News [online]. <https://neurosciencenews.com/huntingtons-embryonic-stem-cell-trial-30944/>

Olson, S.D., Pollock, K., Kambal, A., Cary, W., Mitchell, G.M., Tempkin, J., Stewart, H., McGee, J., Bauer, G., Kim, H.S. and Nolta, J.A. (2012) Genetically engineered mesenchymal stem cells as a proposed therapeutic for Huntington’s disease. Molecular Neurobiology, 45(1), pp.87–98.

Precision Medicine Online (2024) First Huntington’s disease patient dosed with neural stem cell therapy. Inside Precision Medicine [online]. <https://www.insideprecisionmedicine.com/topics/precision-medicine/first-huntingtons-disease-patient-dosed-with-neural-stem-cell-therapy/>

Rossignol, J., Fink, K., Davis, K., Clerc, S., Crane, A., Matchynski, J., Lowrance, S., Bombard, M., Decamp, E., Bavar, S. and Lu, M. (2014) Neural and mesenchymal stem cells in animal models of Huntington’s disease: past experiences and future challenges. Molecular and Cellular Neuroscience, 64, pp.46–60.

Rysankova, I., Sekac, D., Hansikova, H., Kepkova, K.V., Vodicka, P., Vaskovicova, M., Altmanova, M., Juhas, S., Juhasova, J., Taborska, E., Klempir, J., Motlik, J., Klima, J., Eide, L. and Ellederova, Z. (2026) Induced pluripotent stem cells from a transgenic minipig model of Huntington’s disease reveal early metabolic changes. Disease models & mechanisms, 19(6), 052585.

Saudou, F. and Humbert, S. (2016) The Biology of Huntingtin. Neuron, 89(5), pp.910–926.

Schulte, J. and Littleton, J.T. (2011) The biological function of the Huntingtin protein and its relevance to Huntington’s Disease pathology. Current trends in neurology, 5, 65.

Shah, S., Mansour, H.M. and Lucke-Wold, B. (2025) Advances in stem cell therapy for Huntington’s disease: A comprehensive literature review. Cells, 14(1), 42.

Snyder, B.R., Chiu, A.M., Prockop, D.J. and Chan, A.W. (2010) Human multipotent stromal cells (MSCs) increase neurogenesis and decrease atrophy of the striatum in a transgenic mouse model for Huntington’s disease. PLoS ONE, 5(2), e9347.

Supakul, S., Sasaki, Y., Karasawa, K., Kase, Y., Ishikawa, M. and Okano, H. (2026) Modeling striatal development and disease with human pluripotent stem cells. Inflammation and regeneration, 46(32).

Tachibana, C.Y. (2018). Stem-cell culture moves to the third dimension. Nature [online]. <https://www.nature.com/articles/d41586-018-05380-x>

The HD iPSC Consortium (2019) Bioenergetic deficits in Huntington’s disease iPSC-derived neural cells and rescue with glycolytic metabolites. Human Molecular Genetics, 29(11), pp.1757–1771.

Wang, A., Zhang, Y., Deng, H. and Gu, M. (2026) The evolution of iPSC-based cell therapy across autologous and allogeneic pathways. Cell reports. Medicine, 102992.

Wikipedia Contributors (2019) Striatum. Wikipedia [online]. <https://en.wikipedia.org/wiki/Striatum>

Zimmermann, T., Remmers, F., Lutz, B. and Leschik, J. (2016) ESC-Derived BDNF-Overexpressing Neural Progenitors Differentially Promote Recovery in Huntington’s Disease Models by Enhanced Striatal Differentiation. Stem cell reports, 7(4), pp.693–706.