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New gene therapy cures babies with fatal 'Bubble Boy' disease

New gene therapy cures babies with fatal 'Bubble Boy' disease

Researchers find presence of Aire gene in lymph nodes

Researchers find presence of Aire gene in lymph nodes

Researchers discover underlying cause of hereditary muscle disease

Researchers discover underlying cause of hereditary muscle disease

Gene therapy restores hearing in mice with congenital genetic deafness

Gene therapy restores hearing in mice with congenital genetic deafness

Researchers uncover first spontaneous animal model of human hypophosphatasia

Researchers uncover first spontaneous animal model of human hypophosphatasia

Scientists aim to find genetic causes of developmental abnormalities in the vagina and uterus

Scientists aim to find genetic causes of developmental abnormalities in the vagina and uterus

Research team identifies Ashkenazi Jewish founder mutation for Leigh syndrome

Research team identifies Ashkenazi Jewish founder mutation for Leigh syndrome

Geneticists decipher new gene responsible for rare form of hair loss

Geneticists decipher new gene responsible for rare form of hair loss

New form of therapy may stop or reverse progressive vision loss

New form of therapy may stop or reverse progressive vision loss

CNIO researchers link MASTL protein to origin of thrombocytopenia

CNIO researchers link MASTL protein to origin of thrombocytopenia

Newly discovered compound shows potential for treating Parkinson's disease

Newly discovered compound shows potential for treating Parkinson's disease

Majority of Americans’ ancestry can be traced through existing DNA databases

Majority of Americans’ ancestry can be traced through existing DNA databases

New genome editing tool used to heal genetic disease

New genome editing tool used to heal genetic disease

Alnylam submits CTA application to MHRA to initiate Phase 1/2 study of ALN-AAT02

Alnylam submits CTA application to MHRA to initiate Phase 1/2 study of ALN-AAT02

Physical activity may have beneficial effects for people with rare Alzheimer's disease

Physical activity may have beneficial effects for people with rare Alzheimer's disease

Scientists publish study results of AAV therapy product for treatment of RHO-adRP

Scientists publish study results of AAV therapy product for treatment of RHO-adRP

Scientists report novel gene therapy that halts vision loss in canine model of blinding disease

Scientists report novel gene therapy that halts vision loss in canine model of blinding disease

Otsuka’s JINARC approved for treatment of patients with CKD stage 4 ADPKD

Otsuka’s JINARC approved for treatment of patients with CKD stage 4 ADPKD

Research reveals long-term efficacy of drug used to treat common cause of kidney failure

Research reveals long-term efficacy of drug used to treat common cause of kidney failure

Mapping the Genes Responsible for Pluripotency

Mapping the Genes Responsible for Pluripotency