Alpha-1 Antitrypsin Deficiency
Gruntman, Alisha M, Xue, Wen, Flotte, Terence R
Published in Methods in molecular biology (Clifton, N.J.) (2024)
Published in Methods in molecular biology (Clifton, N.J.) (2024)
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Journal Article
Muscle-Directed Delivery of an AAV1 Vector Leads to Capsid-Specific T Cell Exhaustion in Nonhuman Primates and Humans
Gernoux, Gwladys, Gruntman, Alisha M., Blackwood, Meghan, Zieger, Marina, Flotte, Terence R., Mueller, Christian
Published in Molecular therapy (04.03.2020)
Published in Molecular therapy (04.03.2020)
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Journal Article
Gene therapy for alpha-1 antitrypsin deficiency: an update
Pires Ferreira, Debora, Gruntman, Alisha M, Flotte, Terence R
Published in Expert opinion on biological therapy (04.03.2023)
Published in Expert opinion on biological therapy (04.03.2023)
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Journal Article
5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency
Mueller, Christian, Gernoux, Gwladys, Gruntman, Alisha M., Borel, Florie, Reeves, Emer P., Calcedo, Roberto, Rouhani, Farshid N., Yachnis, Anthony, Humphries, Margaret, Campbell-Thompson, Martha, Messina, Louis, Chulay, Jeffrey D., Trapnell, Bruce, Wilson, James M., McElvaney, Noel G., Flotte, Terence R.
Published in Molecular therapy (07.06.2017)
Published in Molecular therapy (07.06.2017)
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Journal Article
Intratracheally administered LNA gapmer antisense oligonucleotides induce robust gene silencing in mouse lung fibroblasts
Shin, Minwook, Chan, Io Long, Cao, Yuming, Gruntman, Alisha M, Lee, Jonathan, Sousa, Jacquelyn, Rodríguez, Tomás C, Echeverria, Dimas, Devi, Gitali, Debacker, Alexandre J, Moazami, Michael P, Krishnamurthy, Pranathi Meda, Rembetsy-Brown, Julia M, Kelly, Karen, Yukselen, Onur, Donnard, Elisa, Parsons, Teagan J, Khvorova, Anastasia, Sontheimer, Erik J, Maehr, René, Garber, Manuel, Watts, Jonathan K
Published in Nucleic acids research (26.08.2022)
Published in Nucleic acids research (26.08.2022)
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Journal Article
Therapeutics: Gene Therapy for Alpha-1 Antitrypsin Deficiency
Gruntman, Alisha M, Flotte, Terence R
Published in Methods in molecular biology (Clifton, N.J.) (01.01.2017)
Published in Methods in molecular biology (Clifton, N.J.) (01.01.2017)
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Journal Article
Modulating immune responses to AAV by expanded polyclonal T-regs and capsid specific chimeric antigen receptor T-regulatory cells
Arjomandnejad, Motahareh, Sylvia, Katelyn, Blackwood, Meghan, Nixon, Thomas, Tang, Qiushi, Muhuri, Manish, Gruntman, Alisha M., Gao, Guangping, Flotte, Terence R., Keeler, Allison M.
Published in Molecular therapy. Methods & clinical development (10.12.2021)
Published in Molecular therapy. Methods & clinical development (10.12.2021)
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Journal Article
Quantification of Total Human Alpha-1 Antitrypsin by Sandwich ELISA
Tang, Qiushi, Gruntman, Alisha M, Flotte, Terence R
Published in Methods in molecular biology (Clifton, N.J.) (01.01.2017)
Published in Methods in molecular biology (Clifton, N.J.) (01.01.2017)
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Journal Article
Improved gene therapy for spinal muscular atrophy in mice using codon-optimized hSMN1 transgene and hSMN1 gene-derived promotor
Xie, Qing, Chen, Xiupeng, Ma, Hong, Zhu, Yunxiang, Ma, Yijie, Jalinous, Leila, Cox, Gerald F, Weaver, Fiona, Yang, Jun, Kennedy, Zachary, Gruntman, Alisha, Du, Ailing, Su, Qin, He, Ran, Tai, Phillip WL, Gao, Guangping, Xie, Jun
Published in EMBO molecular medicine (15.04.2024)
Published in EMBO molecular medicine (15.04.2024)
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Journal Article
Biodistribution and safety of a single rAAV3B-AAT vector for silencing and replacement of alpha-1 antitrypsin in Cynomolgus macaques
Blackwood, Meghan, Gruntman, Alisha M., Tang, Qiushi, Pires-Ferreira, Debora, Reil, Darcy, Kondratov, Oleksandr, Marsic, Damien, Zolotukhin, Sergei, Gernoux, Gwladys, Keeler, Allison M., Mueller, Christian, Flotte, Terence R.
Published in Molecular therapy. Methods & clinical development (14.03.2024)
Published in Molecular therapy. Methods & clinical development (14.03.2024)
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Journal Article
Stability and compatibility of recombinant adeno-associated virus under conditions commonly encountered in human gene therapy trials
Gruntman, Alisha M, Su, Lin, Su, Qin, Gao, Guangping, Mueller, Christian, Flotte, Terence R
Published in Human gene therapy. Part B. Methods (01.04.2015)
Published in Human gene therapy. Part B. Methods (01.04.2015)
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Journal Article
Age-dependent decline in mouse lung regeneration with loss of lung fibroblast clonogenicity and increased myofibroblastic differentiation
Paxson, Julia A, Gruntman, Alisha, Parkin, Christopher D, Mazan, Melissa R, Davis, Airiel, Ingenito, Edward P, Hoffman, Andrew M
Published in PloS one (30.08.2011)
Published in PloS one (30.08.2011)
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Journal Article