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Article

In vivo adenine base editing rescues adrenoleukodystrophy in a humanized mouse model.

Molecular therapy : the journal of the American Society of Gene Therapy - 3 Jul 2024

Gopalappa Ramu, Lee MinYoung, Kim Globinna, Jung Eul Sik, Lee Hanahrae, Hwang Hye-Yeon, Lee Jong Geol, Kim Su Jung, Yoo Hyun Ju, Sung Young Hoon, Kim Daesik, Baek In-Jeoung, Kim Hyongbum Henry

Abstract excerpt

X-linked adrenoleukodystrophy (ALD), an inherited neurometabolic disorder caused by mutations in ABCD1, which encodes the peroxisomal ABC transporter, mainly affects the brain, spinal cord, adrenal glands, and testes. In ALD patients, very-long-chain fatty acids (VLCFAs) fail to enter the peroxisome and undergo subsequent β-oxidation, resulting in their accumulation in the body. It has not been tested whether...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to ATP Binding Cassette Transporter, Subfamily D, Member 1

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Source

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Identifiers

PMID: 38796705
DOI: 10.1016/j.ymthe.2024.05.027
PMCID: PMC11286820

Genes

ABCD1

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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