Lysosomes and their role in Alzheimer’s disease, Duchenne Muscular Dystrophy, Tay-Sachs Disease

U–M researchers reveal how a mutated protein acts as risk factor in Parkinson’s disease Reviewed...

Parents spark breakthrough gene therapy for devastating child disease ignored by big pharma

Exclusive: For more than a century there has been no cure, no treatment and no...

First gene therapy for Tay-Sachs disease successfully given to two children

Two babies have received the first-ever gene therapy for Tay-Sachs disease after over 14 years...

Medical Journal Publication – AAV gene therapy for Tay-Sachs Disease.

Tay-Sachs disease (TSD) is an inherited neurological disorder caused by deficiency of hexosaminidase A (HexA)....

UMass Chan launches Translational Institute for Molecular Therapeutics

Study supported by Blu Genes Published in Nature Medicine Blu Genes Foundation made a commitment...

A family from Marquette is bringing awareness to Tay-Sachs disease

MARQUETTE, Mich. (WLUC) – A rare disease for a one of a kind little boy....

Girl with genetic condition in race against time | CTV News

...

An experimental gene therapy was little Alissa’s only hope. Now, instead of certain death, she faces an uncertain future.

BOSTON — Thomas Feldborg and Daria Rokina set off nearly every afternoon to explore this...

‘Her body was a prison’: How a new gene therapy changed the lives of two Canadian girls

TORONTO — A new gene therapy for an extremely rare disorder has offered two Canadian...

SickKids researchers ‘model’ future of precision medicine for rare inherited disorders

A team of researchers at The Hospital for Sick Children (SickKids) has successfully implemented a...