Last Updated: 09/26/2026
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Found 84 publications
Exa-cel in Children with Transfusion-Dependent β-Thalassemia or Sickle Cell Disease.
Journal: The New England journal of medicine
Published: June 11, 2026
Oligonucleotide Synthesis Errors Are a Source of Untoward Variation in HDR-Mediated Gene Editing.
Journal: Genes
Published: May 12, 2026
Combinatorial base editing couples disease correction with lineage amplification in hematopoietic stem and progenitor cells.
Journal: bioRxiv : the preprint server for biology
Published: April 27, 2026
CRISPR-Cas12a Gene Editing of HBG1 and HBG2 Promoters to Treat β-Thalassemia.
Journal: The New England journal of medicine
Published: April 03, 2026
CRISPR-Cas12a Gene Editing of HBG1 and HBG2 Promoters to Treat Sickle Cell Disease.
Journal: The New England journal of medicine
Published: April 03, 2026
A blueprint to accelerate rare pediatric gene therapy approvals.
Journal: Nature medicine
Published: March 26, 2026
Gene Therapy for Sickle Cell Disease: Practice Recommendations from the American Society for Transplantation and Cellular Therapy and the International Society for Cell & Gene Therapy.
Journal: Transplantation and cellular therapy
Published: March 16, 2026
Synthesis errors in oligonucleotides are propagated into the genome by homology-directed repair.
Journal: bioRxiv : the preprint server for biology
Published: July 17, 2025
Last Updated: 09/26/2026