gene therapy-hemophilia-hemophilia b-fix-factor 9
Gene Therapy Research Offers Transformative Possibilities for People Living With Hemophilia B
Hemophilia B is an ideal target for gene therapy because it’s caused by mutations in a single gene. Gene therapies under investigation aim to provide a working copy of the F9 gene.
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genetic testing-diagnosis-rare disease diagnosis-genetic test-primary immunodeficiency
Rare Diseases
Why Genetic Testing Is the Key to Earlier Rare Disease Diagnoses
For those with a primary immunodeficiency, one of more than 450 rare conditions that affect the immune system, genetic testing holds the key to early diagnosis and appropriate treatment.