Cell and Gene Therapy

Gene Therapy to “reverse” Metabolic disorders! Metabolic disorders are collectively common, frequently severe and in many instances difficult or impossible to treat. Accordingly, there is a compelling need to explore novel therapeutic modalities, including gene therapy, and examine multiple phenotypes where the risks of experimental therapy are outweighed by potential benefits to trial participants. About Gene Therapy Gene therapy is defined as the treatment or cure of human diseases by transfer of nucleic acids (DNA or RNA) to affected cells and it covers a broad range of therapeutic applications from Mendelian disorders to complex metabolic diseases. Most diseases...