Below are the most recent publications written about "alpha 1-Antitrypsin Deficiency" by people in Profiles.
-
Pires Ferreira D, Gruntman AM, Flotte TR. Gene therapy for alpha-1 antitrypsin deficiency: an update. Expert Opin Biol Ther. 2023 03; 23(3):283-291.
-
Ke H, Guay KP, Flotte TR, Gierasch LM, Gershenson A, Hebert DN. Secretion of functional a1-antitrypsin is cell type dependent: Implications for intramuscular delivery for gene therapy. Proc Natl Acad Sci U S A. 2022 08 02; 119(31):e2206103119.
-
Flotte TR. Alpha-1 Antitrypsin Deficiency as a Candidate for Gene Editing. Hum Gene Ther. 2018 08; 29(8):843-844.
-
Song CQ, Wang D, Jiang T, O'Connor K, Tang Q, Cai L, Li X, Weng Z, Yin H, Gao G, Mueller C, Flotte TR, Xue W. In Vivo Genome Editing Partially Restores Alpha1-Antitrypsin in a Murine Model of AAT Deficiency. Hum Gene Ther. 2018 08; 29(8):853-860.
-
Borel F, Tang Q, Gernoux G, Greer C, Wang Z, Barzel A, Kay MA, Shultz LD, Greiner DL, Flotte TR, Brehm MA, Mueller C. Survival Advantage of Both Human Hepatocyte Xenografts and Genome-Edited Hepatocytes for Treatment of a-1 Antitrypsin Deficiency. Mol Ther. 2017 11 01; 25(11):2477-2489.
-
Mueller C, Gernoux G, Gruntman AM, Borel F, Reeves EP, Calcedo R, Rouhani FN, Yachnis A, Humphries M, Campbell-Thompson M, Messina L, Chulay JD, Trapnell B, Wilson JM, McElvaney NG, Flotte TR. 5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency. Mol Ther. 2017 06 07; 25(6):1387-1394.
-
Calcedo R, Somanathan S, Qin Q, Betts MR, Rech AJ, Vonderheide RH, Mueller C, Flotte TR, Wilson JM. Class I-restricted T-cell responses to a polymorphic peptide in a gene therapy clinical trial for a-1-antitrypsin deficiency. Proc Natl Acad Sci U S A. 2017 02 14; 114(7):1655-1659.
-
Gruntman AM. Genotyping Protocol for the Alpha-1 Antitrypsin (PiZ) Mouse Model. Methods Mol Biol. 2017; 1639:61-65.
-
Gruntman AM, Flotte TR. Therapeutics: Gene Therapy for Alpha-1 Antitrypsin Deficiency. Methods Mol Biol. 2017; 1639:267-275.
-
Qiao D, Lange C, Beaty TH, Crapo JD, Barnes KC, Bamshad M, Hersh CP, Morrow J, Pinto-Plata VM, Marchetti N, Bueno R, Celli BR, Criner GJ, Silverman EK, Cho MH. Exome Sequencing Analysis in Severe, Early-Onset Chronic Obstructive Pulmonary Disease. Am J Respir Crit Care Med. 2016 06 15; 193(12):1353-63.