Adeno-associated virus compositions for ARSA gene transfer and methods of use thereof
Provided herein are adeno-associated virus (AAV) compositions that can express an arylsulfatase A (ARSA) polypeptide in a cell, thereby restoring the ARSA gene function. Also provided are methods of using the AAV compositions, and packaging systems for making the AAV compositions.
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| creator | RUBIN, Hillard ST. MARTIN, Thia Baboval SEYMOUR, Albert Barnes |
| description | Provided herein are adeno-associated virus (AAV) compositions that can express an arylsulfatase A (ARSA) polypeptide in a cell, thereby restoring the ARSA gene function. Also provided are methods of using the AAV compositions, and packaging systems for making the AAV compositions. |
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| subjects | BEER BIOCHEMISTRY CHEMISTRY COMPOSITIONS THEREOF CULTURE MEDIA ENZYMOLOGY HUMAN NECESSITIES HYGIENE MEDICAL OR VETERINARY SCIENCE METALLURGY MICROBIOLOGY MICROORGANISMS OR ENZYMES MUTATION OR GENETIC ENGINEERING ORGANIC CHEMISTRY PEPTIDES PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS SPIRITS VINEGAR WINE |
| title | Adeno-associated virus compositions for ARSA gene transfer and methods of use thereof |
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