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
Various Cells Retrovirally Transduced with N -Acetylgalactosoamine-6-Sulfate Sulfatase Correct Morquio Skin Fibroblasts ...
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 with N -Acetylgalactosoamine-6-Sulfate Sulfatase Correct Morquio Skin Fibroblasts ...
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...
Inhibition of matrix metalloproteinases by lung TIMP-1 gene transfer limits monocrotaline-induced pulmonary vascular rem...
Published in Human Gene Therapy
Extracellular matrix dysregulation is key to the development of pulmonary hypertension (PH), suggesting a pivotal role for the proteases that control matrix remodeling. Both hypoxia- and monocrotaline-induced PH are associated with increased protease activity in the distal and proximal pulmonary arteries. However, the role of proteases is not compl...
Clinical protocol. Administration of a replication-deficient adeno-associated virus gene transfer vector expressing the ...
...Published in Human Gene Therapy
Late infantile neuronal ceroid lipofuscinosis (LINCL) is a fatal childhood neurodegenerative lysosomal storage disease with no known therapy. There are estimated to be 200 to 300 children in the United States at any one time with the disease. LINCL is a genetic disease resulting from a deficiency of tripeptidyl peptidase I (TPP-I), a proteolytic en...
α-Fetoprotein gene delivery to the nasal epithelium of nonhuman primates by human parainfluenza viral vectors.
Published in Human gene therapy
Over the last two decades, enormous effort has been focused on developing virus-based gene delivery vectors to target the respiratory airway epithelium as a potential treatment for cystic fibrosis (CF) lung disease. However, amongst other problems, the efficiency of gene delivery to the differentiated airway epithelial cells of the lung has been to...
Zinc-finger nucleases for somatic gene therapy: the next frontier.
Published in Human gene therapy
Zinc-finger nucleases (ZFNs) are a powerful tool that can be used to edit the human genome ad libitum. The technology has experienced remarkable development in the last few years with regard to both the target site specificity and the engineering platforms used to generate zinc-finger proteins. As a result, two phase I clinical trials aimed at knoc...
