Class I-restricted T-cell responses to a polymorphic peptide in a gene therapy clinical trial for α-1-antitrypsin deficiency.

doi:10.1073/pnas.1617726114

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Class I-restricted T-cell responses to a polymorphic peptide in a gene therapy clinical trial for α-1-antitrypsin deficiency.

Proceedings of the National Academy of Sciences of the United States of America - 14 Feb 2017

Calcedo Roberto, Somanathan Suryanarayan, Qin Qiuyue, Betts Michael R, Rech Andrew J, Vonderheide Robert H, Mueller Christian, Flotte Terence R, Wilson James M

Abstract excerpt

Adeno-associated virus (AAV)-mediated gene therapy is currently being pursued as a treatment for the monogenic disorder α-1-antitrypsin (AAT) deficiency. Results from phase I and II studies have shown relatively stable and dose-dependent increases in transgene-derived wild-type AAT after local intramuscular vector administration. In this report we describe the appearance of transgene-specific T-cell responses in...

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Class I-restricted T-cell responses to a polymorphic peptide in a gene therapy clinical trial for α-1-antitrypsin deficiency.

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