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Gene Therapy

Rare Diseases Gene Editing Immune Diseases CRISPR Technology Individualized Treatment Personalized Medicine Antisense Oligonucleotides GBA1 Gene Variants ADA-SCID Beta Thalassaemia ZBTB16 Gene

Scientists Identify ZBTB16-Driven T-Cell Subtype Behind Treatment-Resistant Childhood Leukemia

The marker could be added to routine flow cytometry to flag high-risk T-ALL at diagnosis.

NEJM Study Finds 95% Long-Term Success for ADA-SCID Gene Therapy

Ambroxol Trial Confirms Safety, Brain Delivery and Early Cognitive Benefits in Parkinson’s Dementia