[53] Adeno-associated virus vectors for gene therapy of cystic fibrosis
TR Flotte, BJ Carter - Methods in enzymology, 1998 - Elsevier
Publisher Summary This chapter focuses on cystic fibrosis (CF) that is the most common
genetic disease in North America, with an estimated incidence of one in 2750 live births and
a carrier frequency of approximately 4%. Adeno-associated virus vectors are used for gene
therapy of CF. CF can affect virtually every organ in which there is epithelial ion transport,
the primary clinical manifestations of the disease are caused by exocrine pancreatic
insufficiency and obstructive lung disease. Pancreatic disease is readily compensated for in …
genetic disease in North America, with an estimated incidence of one in 2750 live births and
a carrier frequency of approximately 4%. Adeno-associated virus vectors are used for gene
therapy of CF. CF can affect virtually every organ in which there is epithelial ion transport,
the primary clinical manifestations of the disease are caused by exocrine pancreatic
insufficiency and obstructive lung disease. Pancreatic disease is readily compensated for in …