Adenoassociated Virus Serotype 9-Mediated Gene Therapy for X-Linked Adrenoleukodystrophy-Reference-Cited by-同舟云学术

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Adenoassociated Virus Serotype 9-Mediated Gene Therapy for X-Linked Adrenoleukodystrophy

Published:2015-05 Issue:5 Volume:23 Page:824-834
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Gong Yi,Mu Dakai,Prabhakar Shilpa,Moser Ann,Musolino Patricia,Ren JiaQian,Breakefield Xandra O,Maguire Casey A,Eichler Florian S

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference45 articles.

1. Putative X-linked adrenoleukodystrophy gene shares unexpected homology with ABC transporters;Mosser;Nature,1993

2. Plasma very long chain fatty acids in 3,000 peroxisome disease patients and 29,000 controls;Moser;Ann Neurol,1999

3. Analysis of very long-chain fatty acids using electrospray ionization mass spectrometry;Valianpour;Mol Genet Metab,2003

4. General aspects and neuropathology of X-linked adrenoleukodystrophy;Ferrer;Brain Pathol,2010

5. Pathomechanisms underlying X-adrenoleukodystrophy: a three-hit hypothesis;Singh;Brain Pathol,2010

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1. In vivo adenine base editing rescues adrenoleukodystrophy in a humanized mouse model;Molecular Therapy;2024-07

2. Engineering viral vectors for acoustically targeted gene delivery;Nature Communications;2024-06-10

3. Peripheral Neuropathy in the Adreno-myelo-neuropathy Mouse Model;2024-06-01

4. Therapie der X-chromosomalen Adrenoleukodystrophie;DGNeurologie;2024-04-08

5. Hematopoietic stem cell therapy and ex vivo gene therapy for X-linked adrenoleukodystrophy;Handbook of Clinical Neurology;2024

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