Huntington’s disease (HD) is an inherited neurodegenerative disorder caused by the abnormal expansion of CAG repeats in the Huntingtin gene (HTT). Current treatments focus on symptom relief and comprehensive care. Innovative gene and cell therapy strategies, such as the use of human iPSC-derived striatal neurons (hStrOs) and human umbilical cord mesenchymal stem cells (hUC-MSCs), are being actively researched. These combination therapies aim to target different cellular, epigenetic, and genetic factors, potentially enhancing therapeutic outcomes. Preclinical studies have shown promising results, and the next step is to design clinical trials to assess the safety and efficacy of these innovative strategies.
Key Insights from the review include:
This review highlights the progress in gene and cell therapy for Huntington’s disease, emphasizing the potential of combination therapies to address the complex nature of the disorder. Future research and clinical trials are essential to validate the safety and efficacy of these innovative strategies, which could significantly improve treatment outcomes and ultimately lead to a cure for HD. The work entitled “ Advances in gene and cellular therapeutic approaches for Huntington’s disease ” was published on Protein & Cell (published on Aug. 9, 2024).
Protein & Cell
Systematic review
Not applicable
Advances in gene and cellular therapeutic approaches for Huntington’s disease
9-Aug-2024