Medical research

New gene therapy for 'bubble boy' disease appears effective, safe

A new form of gene therapy for boys with X-linked severe combined immunodeficiency syndrome (SCID-X1), a life-threatening condition also known as "bubble boy" disease, appears to be both effective and safe, according to a ...

Medical research

Friedreich's ataxia: An effective gene therapy in an animal model

The team led by Hélène Puccio, director of research for Inserm at the Institute of Genetics and Molecular and Cellular Biology in close collaboration with Patrick Aubourg's team has demonstrated, in the mice, the efficacy ...

Medical research

Gene therapy trial for 'Bubble boy' disease promising

(Medical Xpress)—Researchers reported promising outcomes data for the first group of boys with X-linked severe combined immunodeficiency syndrome (SCID-X1), a fatal genetic immunodeficiency also known as "bubble boy" disease, ...

Diseases, Conditions, Syndromes

Human neutrophil peptide-1: A new anti-leishmanial drug candidate

Leishmaniasis is a vector borne disease caused by different Leishmania species with different clinical manifestations. Cutaneous leishmaniasis (CL) is endemic and widespread especially among young individuals in Iran. Currently ...

Genetics

No danger of cancer through gene therapy virus

In fall 2012, the European Medicines Agency (EMA) approved the modified adeno-associated virus AAV-LPL S447X as the first ever gene therapy for clinical use in the Western world. uniQure, a Dutch biotech company, had developed ...

Diseases, Conditions, Syndromes

Disease-carrying mosquitos pack twice the punch

An international team of researchers from the University of Notre Dame's Eck Institute for Global Health and Imperial College London has recently published its work on a malaria-filaria co-transmission model, where the same ...

Alzheimer's disease & dementia

Innovative method to treat Alzheimer's in mice

Researchers from the RIKEN Brain Science Institute report that they successfully used a virus vector to restore the expression of a brain protein and improve cognitive functions, in a mouse model of Alzheimer's disease.

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