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Szabo, Gabriela
Manoharan, Pradeep
Lorusso, Anthony
Stem cell gene therapy for fanconi anemia: report from the 1st international Fanconi anemia gene therapy working group meeting.
Approaches to Therapeutic Gene Editing in Alpha-1 Antitrypsin Deficiency.
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Approaches to Therapeutic Gene Editing in Alpha-1 Antitrypsin Deficiency.
Gruntman AM, Xue W, Flotte TR. Approaches to Therapeutic Gene Editing in Alpha-1 Antitrypsin Deficiency. Methods Mol Biol. 2024; 2750:11-17.
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PubMed
subject areas
Alleles
alpha 1-Antitrypsin Deficiency
Gene Editing
Genetic Therapy
Humans
INDEL Mutation
Mutation
authors with profiles
Terence R Flotte MD
Wen Xue PhD
Alisha M Gruntman DVM, PhD