Genetics

New gene therapy for complete color blindness tested in patients

An initial trial in patients indicates that a new genetic treatment for complete color blindness, developed by research groups based in Tübingen and Munich is safe. Preliminary evidence for its efficacy has also been obtained.

Genetics

Benefiting from the national gene vector biorepository

Gene therapy investigators can greatly benefit from the resources and services provided by the National Gene Vector Biorepository (NGVB), housed at the Indiana University School of Medicine. These include 93 unique reagents, ...

Medical research

Long-term hemophilia treatment could lie in patients' own cells

Children (and adults) with hemophilia are slow to form blood clots, so are at constant risk for uncontrolled bleeding. Even when the skin isn't broken, a fall or a simple toe stub can become a serious medical issue: internal ...

Diseases, Conditions, Syndromes

Neuropeptide reduces epileptic seizures in human brain tissue

One challenge facing researchers who study brain diseases is that for understandable reasons it is difficult to obtain human brain tissue for experiments. For that reason, experimental models are used, such as rodent studies, ...

Genetics

Using gene therapy to treat chronic traumatic encephalopathy

A new study shows the feasibility of using gene therapy to treat the progressive neurodegenerative disorder chronic traumatic encephalopathy (CTE). The study, which demonstrated the effectiveness of direct delivery of gene ...

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