Stem cell-based therapy for Huntington's disease

2013 ◽  
Vol 114 (4) ◽  
pp. 754-763 ◽  
Author(s):  
Christof Maucksch ◽  
Elena M. Vazey ◽  
Renee J. Gordon ◽  
Bronwen Connor
Keyword(s):  
Stem Cell ◽  
Huntington's Disease ◽  
Huntington’S Disease ◽  
Cell Based Therapy

scholarly journals Recent Overview of the Use of iPSCs Huntington’s Disease Modeling and Therapy

2020 ◽  
Vol 21 (6) ◽  
pp. 2239 ◽  
Author(s):  
Maria Csobonyeiova ◽  
Stefan Polak ◽  
Lubos Danisovic
Keyword(s):  
Stem Cells ◽  
Stem Cell ◽  
Huntington's Disease ◽  
Huntington’S Disease ◽  
Disease Modeling ◽  
Cell Types ◽  
Cag Repeats ◽  
Neural Cells ◽  
Behavioral Impairment ◽  
Cell Based Therapy

Huntington’s disease (HD) is an inherited, autosomal dominant, degenerative disease characterized by involuntary movements, cognitive decline, and behavioral impairment ending in death. HD is caused by an expansion in the number of CAG repeats in the huntingtin gene on chromosome 4. To date, no effective therapy for preventing the onset or progression of the disease has been found, and many symptoms do not respond to pharmacologic treatment. However, recent results of pre-clinical trials suggest a beneficial effect of stem-cell-based therapy. Induced pluripotent stem cells (iPSCs) represent an unlimited cell source and are the most suitable among the various types of autologous stem cells due to their patient specificity and ability to differentiate into a variety of cell types both in vitro and in vivo. Furthermore, the cultivation of iPSC-derived neural cells offers the possibility of studying the etiopathology of neurodegenerative diseases, such as HD. Moreover, differentiated neural cells can organize into three-dimensional (3D) organoids, mimicking the complex architecture of the brain. In this article, we present a comprehensive review of recent HD models, the methods for differentiating HD–iPSCs into the desired neural cell types, and the progress in gene editing techniques leading toward stem-cell-based therapy.

scholarly journals Stem Cells for Huntington’s Disease (SC4HD): An International Consortium to Facilitate Stem Cell-Based Therapy for Huntington’s Disease

2021 ◽  
pp. 1-6
Author(s):  
Keyword(s):  
Stem Cells ◽  
Stem Cell ◽  
Huntington's Disease ◽  
Huntington’S Disease ◽  
Cell Therapies ◽  
Cell Based Therapy ◽  
Technological Advances ◽  
Regulatory Standards ◽  
Multiple Challenges ◽  
The Brain

Huntington’s disease (HD) research is entering an exciting phase, with new approaches such as huntingtin lowering strategies and cell therapies on the horizon. Technological advances to direct the differentiation of stem cells to desired neural types have opened new strategies for restoring damaged neuronal circuits in HD. However, challenges remain in the implementation of cell therapy approaches for patients suffering from HD. Cell therapies, together with other invasive approaches including allele specific oligonucleotides (ASOs) and viral delivery of huntingtin-lowering agents, require direct delivery of the therapeutic agents locally into the brain or cerebrospinal fluid. Delivering substances directly into the brain is complex and presents multiple challenges, including those related to regulatory requirements, safety and efficacy, surgical instrumentation, trial design, patient profiles, and selection of suitable and sensitive primary and secondary outcomes. In addition, production of clinical grade cell-based medicinal products also requires adherence to regulatory standards with extensive quality control of the protocols and cell products across different laboratories and production centers. Currently, there is no consensus on how best to address these challenges. Here we describe the formation of Stem Cells For Huntington’s Disease (SC4HD: https://www.sc4hd.org/), a network of researchers and clinicians working to develop guidance and greater standardization for the HD field for stem cell based transplantation therapy for HD with a mission to work to develop criteria and guidance for development of a neural intra-cerebral stem cell-based therapy for HD.

scholarly journals Stem cell factor and mesenchymal and neural stem cell transplantation in a rat model of Huntington's disease

2008 ◽  
Vol 37 (3) ◽  
pp. 454-470 ◽  
Author(s):  
Kadiombo Bantubungi ◽  
David Blum ◽  
Laetitia Cuvelier ◽  
Sabine Wislet-Gendebien ◽  
Bernard Rogister ◽  
...  
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Huntington's Disease ◽  
Huntington’S Disease ◽  
Cell Transplantation ◽  
Neural Stem Cell ◽  
Rat Model ◽  
Stem Cell Factor
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