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Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects.

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Bibliographic Details
Title: Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects.
Authors: Colella P; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France. Electronic address: pcolella@stanford.edu., Sellier P; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France., Gomez MJ; Bioinformatics Unit, CNIC, Madrid, Spain., Biferi MG; University Pierre and Marie Curie Paris 6 and INSERM U974, Paris, France., Tanniou G; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France., Guerchet N; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France., Cohen-Tannoudji M; University Pierre and Marie Curie Paris 6 and INSERM U974, Paris, France., Moya-Nilges M; Pasteur Institute, Paris, France., van Wittenberghe L; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France., Daniele N; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France., Gjata B; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France., Krijnse-Locker J; Pasteur Institute, Paris, France., Collaud F; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France., Simon-Sola M; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France., Charles S; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France., Cagin U; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France., Mingozzi F; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France; University Pierre and Marie Curie Paris 6 and INSERM U974, Paris, France; Spark Therapeutics, Philadelphia, PA, USA. Electronic address: federico.mingozzi@sparktx.com.
Source: EBioMedicine [EBioMedicine] 2020 Nov; Vol. 61, pp. 103052. Date of Electronic Publication: 2020 Oct 09.
Publication Type: Journal Article
Journal Info: Publisher: Elsevier B.V Country of Publication: Netherlands NLM ID: 101647039 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2352-3964 (Electronic) Linking ISSN: 23523964 NLM ISO Abbreviation: EBioMedicine Subsets: MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:2352-3964
DOI:10.1016/j.ebiom.2020.103052