CFTR genes and proteins for cystic fibrosis gene therapy

  

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CFTR genes and proteins for cystic fibrosis gene therapy

Invention: CFTR genes and proteins for cystic fibrosis gene therapy

Year    Description
1999Invention patented by John L. Teem on October 21th, 1999. Abstract: The present invention pertains to mutated CFTR genes encoding modified CFTR proteins that result in increased CFTR chloride channel functions in comparison to wildtype CFTR when the CFTR genes are expressed in vivo. Single and multiple amino acid substitutions in a CFTR protein are contemplated in the present invention. The subject invention also concerns modified CFTR proteins encoded by the mutated genes of the invention. The subject invention also concerns methods for increasing CFTR chloride channel activity of a cell by delivering and expressing in the cell a polynucleotide or protein of the invention. The subject invention further concerns methods for treating patients with deficiencies in CFTR function. In one embodiment, the modified CFTR genes of the present invention can be used as a therapeutic agent delivered to CF cells by gene therapy. The modified CFTR genes of the present invention provide CFTR proteins with higher CFTR channel activity than that achievable with the wildtype CFTR gene when expressed in a cell.
Source: selected by the editor from original sources.

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