Genetics

Gene therapy for the treatment of Huntington's disease

Gene therapy targeting the messenger RNA (mRNA) of the mutated huntingtin gene (HTT) can provide long-lasting therapeutic benefit in Huntington's disease after a single administration. An adeno-associated virus (AAV) gene ...

Medical research

Investigators discover new mechanism to boost RNA therapies

Investigators from the Smidt Heart Institute at Cedars-Sinai have identified how biological pacemaker cells—cells that control your heartbeat—can "fight back" against therapies to biologically correct abnormal heartbeat ...

Genetics

Solving the mystery of meningiomas reveals a surprise twist

In solving one mystery—the genetic roots of benign brain tumors called meningiomas—a team of scientists led by Yale researchers stumbled upon an even greater one: How is it possible that two of the mutations linked to ...

Diseases, Conditions, Syndromes

Researchers find controlling element of Huntington's disease

A three molecule complex may be a target for treating Huntington's disease, a genetic disorder affecting the brain. This finding by an international research team including scientists from the German Center for Neurodegenerative ...

Medical research

Researchers replicate human kidney gene changes in mouse model

University of Louisville researchers have replicated the inflammatory gene changes of a human kidney as it progresses from mild to severe diabetic nephropathy, using a mouse model developed by a UofL researcher, according ...

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