Developing Gene Therapy for Mitigating Multisystemic Pathology in Fabry Disease: Proof of Concept in an Aggravated Mouse Model-Reference-Cited by-同舟云学术

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Developing Gene Therapy for Mitigating Multisystemic Pathology in Fabry Disease: Proof of Concept in an Aggravated Mouse Model

Published:2024-07-22 Issue: Volume: Page:
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Boukharov Natalia,Yuan Shipeng,Ruangsirluk Wanida,Ayyadurai Saravanan,Rahman Ashiqur,Rivera-Hernandez Melody¹,Sunkara Shashank,Tonini Kristin,Park Eric Y.H.^ORCID,Deshpande Mugdha,Islam Rizwana

Affiliation:

1. Takeda Pharmaceuticals USA, Inc., Cambridge, Massachusetts, USA.

Publisher

Mary Ann Liebert Inc

Link

https://www.liebertpub.com/doi/pdf/10.1089/hum.2023.222

Reference50 articles.

1. New therapeutic options for lysosomal storage disorders: enzyme replacement, small molecules and gene therapy

2. Pegunigalsidase alfa: a novel, pegylated recombinant alpha-galactosidase enzyme for the treatment of Fabry disease

3. Systemic Treatment of Fabry Disease Using a Novel AAV9 Vector Expressing α-Galactosidase A

4. Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease

5. Lentivirus-mediated gene therapy for Fabry disease

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