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Gene therapy developed for maple syrup urine disease shows promise
MSUD is caused when a mutated form of the BCKDHA, BCKDHB or DBT gene is inherited from both parents. As a result of this mutation, the body is unable to break down certain parts of proteins.
News Medical24d
Study demonstrates successful gene therapy for maple syrup urine disease
MSUD is caused when a mutated form of the BCKDHA, BCKDHB or DBT gene is inherited from both parents. As a result of this mutation, the body is unable to break down certain parts of proteins.
EurekAlert!24d
Gene therapy developed for maple syrup urine disease shows promise, new UMass Chan study reports
MSUD is caused when a mutated form of the BCKDHA, BCKDHB or DBT gene is inherited from both parents. As a result of this mutation, the body is unable to break down certain parts of proteins.
technologynetworks24d
Gene Therapy for Maple Syrup Urine Disease Shows Promise in Animal Study
MSUD is caused when a mutated form of the BCKDHA, BCKDHB or DBT gene is inherited from both parents. As a result of this mutation, the body is unable to break down certain parts of proteins. This ...
GEN20d
AAV Gene Therapy for Maple Syrup Urine Disease Shows Promise
The disease is typically caused by biallelic mutations in genes (branched-chain α-ketoacid dehydrogenase E1α (BCKDHA), E1β (BCKDHB), or dihydrolipoamide branched-chain transacylase (DBT ...