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COL4A5 c.1390G>A
ALS1
DMD c.1332-11868C>G
TP53
Amyotrophic Lateral Sclerosis 1
USH2A c.8559-2A>G
Cystic Fibrosis
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News & Insights
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AI-Empowered Breakthroughs in Genetic Disease Research: Industry Pain Points and RDDC Solutions
November 26, 2025
Industry Insights
Hemophilia & Gene Therapy
Industry Insights
Hemophilia: A Beacon of Hope from Gene Mutation to Gene Therapy
November 01, 2025
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Phenylketonuria (PKU)
Industry Insights
PKU Treatment Breakthrough: Mechanism Analysis from Cofactor Replacement (Sephience) to Gene Silencing (GS1168)
September 30, 2025
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