Intrathecal AAV9/AP4M1 gene therapy for hereditary spastic paraplegia 50 shows safety and efficacy in preclinical studies-Reference-Cited by-同舟云学术

Intrathecal AAV9/AP4M1 gene therapy for hereditary spastic paraplegia 50 shows safety and efficacy in preclinical studies

Published:2023-05-15 Issue:10 Volume:133 Page:
ISSN:1558-8238
Container-title:Journal of Clinical Investigation
language:en
Short-container-title:

Author:

Chen Xin^ORCID,Dong Thomas,Hu Yuhui,De Pace Raffaella^ORCID,Mattera Rafael^ORCID,Eberhardt Kathrin,Ziegler Marvin^ORCID,Pirovolakis Terry,Sahin Mustafa^ORCID,Bonifacino Juan S.^ORCID,Ebrahimi-Fakhari Darius^ORCID,Gray Steven J.^ORCID

Funder

CureSPG50 Foundation

Publisher

American Society for Clinical Investigation

Subject

General Medicine

Link

https://www.jci.org/articles/view/164575/files/pdf

Reference61 articles.

1. Defining the clinical, molecular and imaging spectrum of adaptor protein complex 4-associated hereditary spastic paraplegia;Ebrahimi-Fakhari;Brain,2020

2. Ebrahimi-Fakhari D, et al. AP-4-Associated hereditary spastic paraplegia. In: Adam MP, et al., eds. GeneReviews. University of Washington; 2018:1993–2023. https://www.ncbi.nlm.nih.gov/books/NBK535153/ Accessed May 3, 2023

3. Mutation in the AP4M1 Gene Provides a Model for Neuroaxonal Injury in Cerebral Palsy

4. Systematic Analysis of Brain MRI Findings in Adaptor Protein Complex 4–Associated Hereditary Spastic Paraplegia

5. AP-4, a Novel Protein Complex Related to Clathrin Adaptors

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