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Targeting Age-Related Neurodegenerative Diseases by AAV-Mediated Gene Therapy.
Adv Exp Med Biol. 2021; 1286:213-223.AE

Abstract

Age-related neurodegenerative diseases have detrimental consequences on health of many patients and result in mortality. The current treatment options are limited and usually fail to correct the underlying pathology. AAV-based gene therapies have proved to be safe based on the data available on clinical trials for several monogenic diseases. Therefore, such therapies can pave the way to treat neurodegenerative diseases likes Alzheimer's disease (AD), Parkinson's disease (PD), and amyotrophic lateral sclerosis (ALS). Here, the advantages of AAV-based gene therapies are discussed with emphasis on efforts of developing novel capsids with superior therapeutic efficacy. Furthermore, the results of clinical trials on AD, PD, and ALS are summarized.

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Publisher Full Text (DOI)

Authors+Show Affiliations

Cagin U
INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, Evry, France. ucagin@genethon.fr.

MeSH

Alzheimer DiseaseAmyotrophic Lateral SclerosisGenetic TherapyHumansNeurodegenerative DiseasesParkinson Disease

Pub Type(s)

Journal Article

Language

eng

PubMed ID

33725356

Citation

Cagin, Umut. "Targeting Age-Related Neurodegenerative Diseases By AAV-Mediated Gene Therapy." Advances in Experimental Medicine and Biology, vol. 1286, 2021, pp. 213-223.
Cagin U. Targeting Age-Related Neurodegenerative Diseases by AAV-Mediated Gene Therapy. Adv Exp Med Biol. 2021;1286:213-223.
Cagin, U. (2021). Targeting Age-Related Neurodegenerative Diseases by AAV-Mediated Gene Therapy. Advances in Experimental Medicine and Biology, 1286, 213-223. https://doi.org/10.1007/978-3-030-55035-6_15
Cagin U. Targeting Age-Related Neurodegenerative Diseases By AAV-Mediated Gene Therapy. Adv Exp Med Biol. 2021;1286:213-223. PubMed PMID: 33725356.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Targeting Age-Related Neurodegenerative Diseases by AAV-Mediated Gene Therapy. A1 - Cagin,Umut, PY - 2021/3/16/entrez PY - 2021/3/17/pubmed PY - 2021/3/19/medline KW - AAV KW - CNS KW - Capsid KW - Central nervous system KW - Neurodegeneration gene therapy KW - Neurodegenerative diseases SP - 213 EP - 223 JF - Advances in experimental medicine and biology JO - Adv Exp Med Biol VL - 1286 N2 - Age-related neurodegenerative diseases have detrimental consequences on health of many patients and result in mortality. The current treatment options are limited and usually fail to correct the underlying pathology. AAV-based gene therapies have proved to be safe based on the data available on clinical trials for several monogenic diseases. Therefore, such therapies can pave the way to treat neurodegenerative diseases likes Alzheimer's disease (AD), Parkinson's disease (PD), and amyotrophic lateral sclerosis (ALS). Here, the advantages of AAV-based gene therapies are discussed with emphasis on efforts of developing novel capsids with superior therapeutic efficacy. Furthermore, the results of clinical trials on AD, PD, and ALS are summarized. SN - 0065-2598 UR - https://www.unboundmedicine.com/medline/citation/33725356/Targeting_Age_Related_Neurodegenerative_Diseases_by_AAV_Mediated_Gene_Therapy_ DB - PRIME DP - Unbound Medicine ER -