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Stem cells for Huntington’s disease (SC4HD): an international consortium to facilitate stem cell-based therapy for Huntington’s disease

Badin, Romina Aron, Bachoud-Levi, Anne-Catherine, Bauer, Gerhard, Busse-Morris, Monica ORCID: https://orcid.org/0000-0002-5331-5909, Canais, Josep M., Capetian, Philipp, Cattaneo, Elena, Chen, Jefferson, Cozzi, Emanule, Ellendorova, Zdenka, Goldman, Steven A., Gray, WIlliam ORCID: https://orcid.org/0000-0001-7595-8887, Lai, Liangxue, Li, Meng ORCID: https://orcid.org/0000-0002-4803-4643, Morenkova, Anna, Pan, Guanglin, Pei, Zhong, Unai Perpina, Martin, Perrier, Anselme, Reidling, John C., Rosser, Anne E. ORCID: https://orcid.org/0000-0002-4716-4753, Song, Jiwhan, Thompson, Leslie M. and Wheelock, Vicki 2021. Stem cells for Huntington’s disease (SC4HD): an international consortium to facilitate stem cell-based therapy for Huntington’s disease. Journal of Huntington's Disease 10 (2) , pp. 221-226. 10.3233/JHD-210473

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Abstract

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.

Item Type: Article
Status: Published
Schools: Medicine
MRC Centre for Neuropsychiatric Genetics and Genomics (CNGG)
Additional Information: This is an Open Access article distributed under the terms of the Creative Commons Attribution-NonCommercial License (CC BY-NC 4.0)
Publisher: IOS Press
ISSN: 1879-6397
Date of First Compliant Deposit: 25 February 2022
Date of Acceptance: 26 February 2021
Last Modified: 29 Nov 2022 11:00
URI: https://orca.cardiff.ac.uk/id/eprint/147831

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