Various Cells Retrovirally Transduced withN-Acetylgalactosoamine-6-Sulfate Sulfatase Correct Morquio Skin FibroblastsIn ...
Published in Human Gene Therapy
Various cells retrovirally transduced with N-acetylgalactosoamine-6-sulfate sulfatase correct Morquio skin fibroblasts i...
Published in Human Gene Therapy
Gene therapy may provide a long-term approach to the treatment of mucopolysaccharidoses. As a first step toward the development of an effective gene therapy for mucopolysaccharidosis type IVA (Morquio syndrome), a recombinant retroviral vector, LGSN, derived from the LXSN vector, containing a full-length human wildtype N-acetylgalactosamine-6-sulfa...
Various Cells Retrovirally Transduced withN-Acetylgalactosoamine-6-Sulfate Sulfatase Correct Morquio Skin FibroblastsIn ...
Published in Human Gene Therapy
Various Cells Retrovirally Transduced withN-Acetylgalactosoamine-6-Sulfate Sulfatase Correct Morquio Skin FibroblastsIn ...
Published in Human Gene Therapy
Electrotransfer of the Full-Length Dog Dystrophin into Mouse and Dystrophic Dog Muscles
Published in Human Gene Therapy
Duchenne muscular dystrophy (DMD) is an X-linked genetic disease characterized by the absence of dystrophin (427 kDa). An approach to eventually restore this protein in patients with DMD is to introduce into their muscles a plasmid encoding dystrophin cDNA. Because the phenotype of the dystrophic dog is closer to the human phenotype than is the mdx...
