Zobrazeno 1 - 10
of 683
pro vyhledávání: '"P B McCray"'
Autor:
Katarina Kulhankova, Soumba Traore, Xue Cheng, Hadrien Benk-Fortin, Stéphanie Hallée, Mario Harvey, Joannie Roberge, Frédéric Couture, Sajeev Kohli, Thomas J. Gross, David K. Meyerholz, Garrett R. Rettig, Bernice Thommandru, Gavin Kurgan, Christine Wohlford-Lenane, Dennis J. Hartigan-O’Connor, Bradley P. Yates, Gregory A. Newby, David R. Liu, Alice F. Tarantal, David Guay, Paul B. McCray
Publikováno v:
Nature Communications, Vol 14, Iss 1, Pp 1-16 (2023)
Abstract Gene editing strategies for cystic fibrosis are challenged by the complex barrier properties of airway epithelia. We previously reported that the amphiphilic S10 shuttle peptide non-covalently combined with CRISPR-associated (Cas) ribonucleo
Externí odkaz:
https://doaj.org/article/8ac54f1857804245a0069d9ef38b1f91
Autor:
Ashley L. Cooney, Christian M. Brommel, Soumba Traore, Gregory A. Newby, David R. Liu, Paul B. McCray, Patrick L. Sinn
Publikováno v:
Frontiers in Genome Editing, Vol 5 (2023)
Considerable effort has been devoted to developing adeno-associated virus (AAV)-based vectors for gene therapy in cystic fibrosis (CF). As a result of directed evolution and capsid shuffling technology, AAV capsids are available with widespread tropi
Externí odkaz:
https://doaj.org/article/9022961518b6498c9920e9f7bf41f264
Autor:
Lei Lei, Soumba Traore, Guillermo S. Romano Ibarra, Philip H. Karp, Tayyab Rehman, David K. Meyerholz, Joseph Zabner, David A. Stoltz, Patrick L. Sinn, Michael J. Welsh, Paul B. McCray Jr., Ian M. Thornell
Publikováno v:
The Journal of Clinical Investigation, Vol 133, Iss 20 (2023)
The volume and composition of a thin layer of liquid covering the airway surface defend the lung from inhaled pathogens and debris. Airway epithelia secrete Cl– into the airway surface liquid through cystic fibrosis transmembrane conductance regula
Externí odkaz:
https://doaj.org/article/2cd2777105f44a3382766c3e36dc6f28
Autor:
Matthew D. Strub, Shyam Ramachandran, Dmitri Y. Boudko, Ella A. Meleshkevitch, Alejandro A. Pezzulo, Aravind Subramanian, Arthur Liberzon, Robert J. Bridges, Paul B. McCray Jr.
Publikováno v:
CPT: Pharmacometrics & Systems Pharmacology, Vol 11, Iss 2, Pp 240-251 (2022)
Abstract Cystic fibrosis (CF) is a lethal autosomal recessive disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The common ΔF508‐CFTR mutation results in protein misfolding and proteasomal degradat
Externí odkaz:
https://doaj.org/article/118b9841113341758b26c7d71659a1be
Autor:
Jose M. Honrubia, Javier Gutierrez-Álvarez, Alejandro Sanz-Bravo, Ezequiel González-Miranda, Diego Muñoz-Santos, Carlos Castaño-Rodriguez, Li Wang, Marta Villarejo-Torres, Jorge Ripoll-Gómez, Ana Esteban, Raul Fernandez-Delgado, Pedro José Sánchez-Cordón, Juan Carlos Oliveros, Stanley Perlman, Paul B. McCray, Isabel Sola, Luis Enjuanes
Publikováno v:
mBio, Vol 14, Iss 1 (2023)
ABSTRACT Coronaviruses (CoVs) of genera α, β, γ, and δ encode proteins that have a PDZ-binding motif (PBM) consisting of the last four residues of the envelope (E) protein (PBM core). PBMs may bind over 400 cellular proteins containing PDZ domain
Externí odkaz:
https://doaj.org/article/748cab695f5f46f68ab0f4651f18e0e3
Publikováno v:
BMC Medical Genomics, Vol 14, Iss 1, Pp 1-11 (2021)
Abstract Background We previously reported that expression of a miR-138 mimic or knockdown of SIN3A in primary cultures of cystic fibrosis (CF) airway epithelia increased ΔF508-CFTR mRNA and protein levels, and partially restored CFTR-dependent chlo
Externí odkaz:
https://doaj.org/article/7b9ca34f3d994a4680d99ff464e34bc0
Autor:
Ashley L. Cooney, Andrew L. Thurman, Paul B. McCray, Jr., Alejandro A. Pezzulo, Patrick L. Sinn
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 25, Iss , Pp 293-301 (2021)
Life-long expression of a gene therapy agent likely requires targeting stem cells. Here we ask the question: does viral vector transduction or ectopic expression of a therapeutic transgene preclude airway stem cell function? We used a lentiviral vect
Externí odkaz:
https://doaj.org/article/81f681dea87b44bdb14b49d961933705
Autor:
Laura I. Marquez Loza, Ashley L. Cooney, Qian Dong, Christoph O. Randak, Stefano Rivella, Patrick L. Sinn, Paul B. McCray, Jr.
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 21, Iss , Pp 94-106 (2021)
Despite significant advances in cystic fibrosis (CF) treatments, a one-time treatment for this life-shortening disease remains elusive. Stable complementation of the disease-causing mutation with a normal copy of the CF transmembrane conductance regu
Externí odkaz:
https://doaj.org/article/d0519b4aa458485db73ff7f204fe6471
Autor:
Rachel A. Hodos, Matthew D. Strub, Shyam Ramachandran, Ella A. Meleshkevitch, Dmitri Y. Boudko, Robert J. Bridges, Joel T. Dudley, Paul B. McCray Jr.
Publikováno v:
CPT: Pharmacometrics & Systems Pharmacology, Vol 10, Iss 5, Pp 500-510 (2021)
Abstract Rare diseases affect 10% of the first‐world population, yet over 95% lack even a single pharmaceutical treatment. In the present age of information, we need ways to leverage our vast data and knowledge to streamline therapeutic development
Externí odkaz:
https://doaj.org/article/35af72e80c624b9c9410aa56577a0eb6
Publikováno v:
Scientific Reports, Vol 10, Iss 1, Pp 1-16 (2020)
Abstract Cystic fibrosis (CF), caused by mutations to CFTR, leads to severe and progressive lung disease. The most common mutant, ΔF508-CFTR, undergoes proteasomal degradation, extinguishing its anion channel function. Numerous in vitro intervention
Externí odkaz:
https://doaj.org/article/7df26fe9ff194bfba3a28caf8cd5a311