Published April 28, 2026 | Version v1
Preprint Open

ABCD1 Gene Therapy for X-linked Adrenoleukodystrophy Dementia

Authors/Creators

  • 1. MedicOath

Description

ABCD1 scores 0.88 in Open Targets and maps to peroxisome pathway (KEGG: hsa04146) and ABC transporters (hsa02010). ClinVar shows multiple ABCD1 variants. Gene therapy could restore peroxisomal function and prevent demyelination-associated cognitive decline in X-ALD.

Notes

IPFS: QmboDz3EDajh4JNhxyiTvDS9z7s2ZsDbidVzFiMRjic6Bx. TX: 0xe9b4c6dd133a2ea725eefee7c3f2c3ec8359650b42942b86ce46e98fa559bf53. CC0.

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