Gene Therapy Progress and Prospects: Alpha-1 antitrypsin
Autor: | K L Brigham, A A Stecenko |
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Rok vydání: | 2003 |
Předmět: |
congenital
hereditary and neonatal diseases and abnormalities Genetic enhancement Transgene Genetic Vectors Mutant Biology Gene delivery In vivo alpha 1-Antitrypsin Deficiency Gene expression Genetics Animals Humans Vector (molecular biology) Lung Molecular Biology Gene Gene Transfer Techniques DNA Genetic Therapy Dependovirus Nasal Mucosa Liver Pulmonary Emphysema alpha 1-Antitrypsin Liposomes Immunology Cancer research Molecular Medicine |
Zdroj: | Gene Therapy. 10:95-99 |
ISSN: | 1476-5462 0969-7128 |
Popis: | Over the 2 years covered here, there has been one clinical study in which a normal alpha-1 antitrypsin (AAT) gene was delivered to the nasal epithelium of AAT-deficient subjects using plasmid-liposome complexes; a second study using an adeno-associated vector should begin soon. Although progress in clinical studies has been slow, advances in both viral and nonviral vector designs show considerable promise. Strategies that combine liposome technology with imaginative vector design may permit long-term expression of a normal transgene that is sufficient to achieve therapeutic serum AAT concentrations. While reproducing the normal physiology by targeting normal AAT gene expression to the liver is logical, local expression in lung cells may be less demanding of the technology and offers therapeutic benefits that are produced neither by AAT protein therapy nor by AAT gene therapy targeted to the liver. Developing technologies may permit direct correction of the mutant AAT gene using innovative approaches to in vivo gene repair. |
Databáze: | OpenAIRE |
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