Search Results - "Gruntman, Alisha"

Serum Western Blot for the Detection of a c-Myc Protein Tag in Non-human Primates and Mice by Blackwood, Meghan, Tang, Qiushi, Gruntman, Alisha M

“…This protocol allows for the detection of a c-Myc tag on alpha-1 antitrypsin (AAT) delivered to species that already have endogenous AAT such as non-human…”
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The rapidly evolving state of gene therapy by Gruntman, Alisha M., Flotte, Terence R.

“…Gene therapy is emerging as a viable option for clinical therapy of monogenic disorders and other genetically defined diseases, with approved gene therapies…”
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Approaches to Therapeutic Gene Editing in Alpha-1 Antitrypsin Deficiency by Gruntman, Alisha M, Xue, Wen, Flotte, Terence R

“…Five distinct gene therapy approaches have been developed for treating AATD. These approaches include knockout of the mutant (PiZ) allele by introduction of…”
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Alpha-1 Antitrypsin Deficiency by Gruntman, Alisha M, Xue, Wen, Flotte, Terence R

“…Alpha-1 antitrypsin (AAT) deficiency is a common monogenic disorder in which there is a strong founder effect of a single missense mutation in SERPINA1, the…”
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Muscle-Directed Delivery of an AAV1 Vector Leads to Capsid-Specific T Cell Exhaustion in Nonhuman Primates and Humans by Gernoux, Gwladys, Gruntman, Alisha M., Blackwood, Meghan, Zieger, Marina, Flotte, Terence R., Mueller, Christian

“…With the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) approvals for Zolgensma, Luxturna, and Glybera, recombinant adeno-associated…”
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Gene Therapy and the Use of Animal Models: Why Mice Alone Are Not Sufficient by Gruntman, Alisha M, Flotte, Terence R

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Gene therapy for alpha-1 antitrypsin deficiency: an update by Pires Ferreira, Debora, Gruntman, Alisha M, Flotte, Terence R

“…Altering the human genetic code has been explored since the early 1990s as a definitive answer for the treatment of monogenic and acquired diseases which do…”
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Genotyping Protocol for the Alpha-1 Antitrypsin (PiZ) Mouse Model by Gruntman, Alisha M

“…The most common alpha-1 antitrypsin (AAT) mutant variant is a missense mutation (E342K), commonly referred to as PiZ. A transgenic mouse model exists that…”
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5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency by 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.

“…Alpha-1 antitrypsin deficiency is a monogenic disorder resulting in emphysema due principally to the unopposed effects of neutrophil elastase. We previously…”
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Retro-Orbital Venous Sinus Delivery of rAAV9 Mediates High-Level Transduction of Brain and Retina Compared with Temporal Vein Delivery in Neonatal Mouse Pups by Gruntman, Alisha M, Su, Lin, Flotte, Terence R

“…In order to pursue a clinical gene therapy for a human neurologic disease, it is often necessary to perform proof-of-concept trials in mouse models of that…”
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Intratracheally administered LNA gapmer antisense oligonucleotides induce robust gene silencing in mouse lung fibroblasts by 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

“…Abstract The lung is a complex organ with various cell types having distinct roles. Antisense oligonucleotides (ASOs) have been studied in the lung, but it has…”
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Therapeutics: Gene Therapy for Alpha-1 Antitrypsin Deficiency by Gruntman, Alisha M, Flotte, Terence R

“…This review seeks to give an overview of alpha-1 antitrypsin deficiency, including the different disease phenotypes that it encompasses. We then describe the…”
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Modulating immune responses to AAV by expanded polyclonal T-regs and capsid specific chimeric antigen receptor T-regulatory cells by Arjomandnejad, Motahareh, Sylvia, Katelyn, Blackwood, Meghan, Nixon, Thomas, Tang, Qiushi, Muhuri, Manish, Gruntman, Alisha M., Gao, Guangping, Flotte, Terence R., Keeler, Allison M.

“…Immune responses to adeno-associated virus (AAV) capsids limit the therapeutic potential of AAV gene therapy. Herein, we model clinical immune responses by…”
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Quantification of Total Human Alpha-1 Antitrypsin by Sandwich ELISA by Tang, Qiushi, Gruntman, Alisha M, Flotte, Terence R

“…In this chapter we describe an enzyme-linked immunosorbent assay (ELISA) to quantitatively measure human alpha-1 antitrypsin (AAT) protein levels in serum,…”
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Improved gene therapy for spinal muscular atrophy in mice using codon-optimized hSMN1 transgene and hSMN1 gene-derived promotor by 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

“…Physiological regulation of transgene expression is a major challenge in gene therapy. Onasemnogene abeparvovec ( Zolgensma ® ) is an approved adeno-associated…”
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Biodistribution and safety of a single rAAV3B-AAT vector for silencing and replacement of alpha-1 antitrypsin in Cynomolgus macaques by 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.

“…Alpha-1 antitrypsin deficiency (AATD) is characterized by both chronic lung disease due to loss of wild-type AAT (M-AAT) antiprotease function and liver…”
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Limb Perfusion Delivery of a rAAV1 Alpha-1 Antitrypsin Vector in Non-Human Primates Is Safe but Insufficient for Therapy by Pires-Ferreira, Debora, Reil, Darcy, Tang, Qiushi, Blackwood, Meghan, Gallagher, Thomas, Keeler, Allison M, Chichester, Jessica A, Vyhnal, Kristin K, Lindborg, Jane A, Benson, Janet, Fu, Dongtao, Flotte, Terence R, Gruntman, Alisha M

“…α-1 antitrypsin (AAT) deficiency is an inherited, genetic condition characterized by reduced serum levels of AAT and increased risk of developing emphysema and…”
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Stability and compatibility of recombinant adeno-associated virus under conditions commonly encountered in human gene therapy trials by Gruntman, Alisha M, Su, Lin, Su, Qin, Gao, Guangping, Mueller, Christian, Flotte, Terence R

“…Recombinant adeno-associated virus (rAAV) vectors are rapidly becoming the first choice for human gene therapy studies, as clinical efficacy has been…”
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Age-dependent decline in mouse lung regeneration with loss of lung fibroblast clonogenicity and increased myofibroblastic differentiation by Paxson, Julia A, Gruntman, Alisha, Parkin, Christopher D, Mazan, Melissa R, Davis, Airiel, Ingenito, Edward P, Hoffman, Andrew M

“…While aging leads to a reduction in the capacity for regeneration after pneumonectomy (PNX) in most mammals, this biological phenomenon has not been…”
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Age dependence of lung mesenchymal stromal cell dynamics following pneumonectomy by Paxson, Julia A, Gruntman, Alisha M, Davis, Airiel M, Parkin, Christopher M, Ingenito, Edward P, Hoffman, Andrew M

“…Aging is a critical determinant of regenerative capacity in many organ systems, but it remains unresolved in the lung. This study examines murine lung cell…”
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