Article Abstract:
A study by Maheshri and group extend error prone PCR and a staggered extension process into the realm of recombination (adeno-associated virus) AAV vectors which not only adds to a rapid growing vector tool kit but also sheds light on structural aspects of the AAV caspid. The study by Maheshri and group is a clear step towards the ultimate goal of engineering AAV vectors tailored to individual patient or disease profiles.
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Article Abstract:
Adeno-associated virus (AAV) vectors that contain single-stranded DNA can efficiently transduce cells in vivo and are shown to target homologous chromosomal sequences in cultured cells. In order to correct the naturally occurring GusB gene mutation that was responsible for murine mucopolysaccharidosis type VII(super 2) AAV genetargeting vectors are used.
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Article Abstract:
Hepatic gene correction by adeno-associated Virus (AAV) vectors in vivo is demonstrated, using both a mutant LacZ reporter mouse and a murine model of mucopolysaccharidosis type VII. These studies provide a foundation for the development of AAV vector systems for targeted gene therapy in humans.
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