.png){kind=icon}
.png){kind=icon}
Return
Safety considerations of gene-based therapies for Alzheimer's disease
.png){kind=icon}
OA
AI
E
G
M
J
S
M
J
H
DOI：10.1002/alz.71515.png)
.png){kind=icon}
.png){kind=icon}
Share.png){kind=icon}
Save
Abstract
En 中文
Gene-based therapies show increasing promise for the treatment of neurologic disease. In 2016, nusinersen, an RNA-based therapy, was approved for children with spinal muscular atrophy (SMA). Over 200 clinical trials have utilized gene therapy approaches for a host of neurodegenerative and neuromuscular disorders, including Alzheimer's disease (AD).1 Delivering gene-based therapies to the central nervous system (CNS) has raised safety concerns. As clinical trials utilizing gene therapy paradigms are undertaken and as approved therapies translate into clinics, a better understanding of safety issues and monitoring parameters is needed. We describe current therapeutic approaches for AD using gene-based therapies such as adeno-associated virus gene therapy and RNA interference by antisense oligonucleotides (ASOs) and undertake a review of medical safety considerations for existing AD gene therapy trials. We identify challenges in analyzing medical safety data currently available and provide guidelines for making this information more accessible and interpretable for the future.
Keywords:
AAV
adeno-associated virus
Alzheimer's disease
central nervous system
clinical trials
gene therapy
gene-based therapy
genetic mutation
medical safety
medical safety data
mild cognitive impairment
neurodegenerative disease
neurodegenerative disorders
neurotoxicity
safety and risk assessment
AI Summary
Key information extracted from the uploaded paper, including a brief overview, abstract, background, key highlights, visual analysis, and future outlook.
Journal
A
IF:
0
Papers:
511
Citations:
0