Clinical trial perspective for adult and juvenile Huntington’s disease using genetically-engineered mesenchymal stem cells
Peter Deng
Audrey Torrest
Kari Pollock
Heather Dahlenburg
Geralyn Annett
Jan A. Nolta
Kyle D. Fink
摘要：Progress to date from our group and others indicate that using genetically-engineered mesenchymal stem cells (MSC) to secrete brain-derived neurotrophic factor (BDNF) supports our plan to submit an Investigational New Drug application to the Food and Drug Administration for the future planned Phase 1 safety and tolera-bility trial of MSC/BDNF in patients with Huntington’s disease (HD). There are also potential applications of this approach beyond HD. Our biological delivery system for BDNF sets the precedent for adult stem cell ther-apy in the brain and could potentially be modiifed for other neurodegenerative disorders such as amyotrophic lateral sclerosis (ALS), spinocerebellar ataxia (SCA), Alzheimer’s disease, and some forms of Parkinson’s dis-ease. The MSC/BDNF product could also be considered for studies of regeneration in traumatic brain injury, spinal cord and peripheral nerve injury. This work also provides a platform for our future gene editing studies, since we will again use MSCs to deliver the needed molecules into the central nervous system.
机标关键词：amyotrophic lateral sclerosisneurodegenerative disordersperipheral nerve injurytraumatic brain injurymesenchymal stem cellscentral nervous systemneurotrophic factorDrug Administration
论文发表日期：2016-01-01
在线出版日期：2025-08-15（本平台首次上网日期，不代表文献的发表时间）
页数：4( 702-705 )
英文信息