Editorial for Special Issue on Gene Therapy of Rare Diseases

Cord Brakebusch

PMC · DOI:10.3390/cells15060504·March 12, 2026

Editorial for Special Issue on Gene Therapy of Rare Diseases

Cord Brakebusch

PDF

Open Access

Abstract

Linked entities

Genes, proteins, chemicals, diseases, species, mutations and cell lines named across the full text — each resolved to its canonical identifier and authoritative record.

Genes1

Galc

Proteins1

Species2

Mus musculus(house mouse · species)Homo sapiens(human · species)

Chemicals1

AAV9

Diseases14

beta-thalassemia rare diseases injury to alpha1-antitrypsin disease spinal muscular atrophy metachromatic leukodystrophy cerebral adrenoleukodystrophy Diamond-Blackfan Anemia lysosomal storage disorder Globoid Cell Leukodystrophy hemophilia A and B.sickle cell anemia Wilson disease of the liver

Peer Reviews

No public reviews on file for this paper yet. If you reviewed it on a platform where reviews are public (OpenReview, ICLR, NeurIPS, ICML), you can paste yours below so the community can read it here.

Videos

No videos yet. Explain this paper in a talk, walkthrough, or lecture? Add one.

Taxonomy

TopicsVirus-based gene therapy research · Lysosomal Storage Disorders Research · CRISPR and Genetic Engineering