Translation of gene therapy strategies for amyotrophic lateral sclerosis
Trends Mol Med
.
2022 Sep;28(9):795-796.
doi: 10.1016/j.molmed.2022.07.001.
Epub 2022 Jul 28.
Authors
Tyler G Demarest
1
,
Maria Grazia Biferi
2
Affiliations
1
Spark Therapeutics, Philadelphia, PA 19104, USA.
2
Spark Therapeutics, Philadelphia, PA 19104, USA. Electronic address: maria.graziabiferi@sparktx.com.
PMID:
35909024
DOI:
10.1016/j.molmed.2022.07.001
No abstract available
Keywords:
AAV; ALS; ASO; clinical trials (CT); gene therapy.
MeSH terms
Amyotrophic Lateral Sclerosis*
Genetic Therapy
Humans