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Preclinical Development of Antisense Oligonucleotides to Rescue Aberrant Splicing Caused by an Ultrarare ABCA4 Variant in a Child with Early-Onset Stargardt Disease

1
1. Vibhor 26 Jul 2026
  
  in read_only_demo_group
  
  Preclinical Development of Antisense Oligonucleotides to Rescue Aberrant Splicing Caused by an Ultrarare ABCA4 Variant in a Child with Early-Onset Stargardt Disease
  
  PMID: 38607040
  
  Gene: ABCA4
  
  HGNC ID: 34
  
  Clin Gen General HGNC: 34 Gene:ABCA4 automated_hgnc:34 automated_hgnc_source:Gene_Name ClinGen ABCA4 Annotations
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ClinGen ABCA4 Annotations

automated_hgnc_source:Gene_Name

automated_hgnc:34

General

HGNC: 34

Clin Gen

Gene:ABCA4

Annotators

Vibhor

URL

pmc.ncbi.nlm.nih.gov/articles/PMC11011354/