Zobrazeno 1 - 10
of 36
pro vyhledávání: '"Ashley L. Cooney"'
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:
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
Publikováno v:
Frontiers in Genome Editing, Vol 3 (2022)
Pulmonary surfactant is critically important to prevent atelectasis by lowering the surface tension of the alveolar lining liquid. While respiratory distress syndrome (RDS) is common in premature infants, severe RDS in term and late preterm infants s
Externí odkaz:
https://doaj.org/article/fcdff47321004f249cd2be27366c32cb
Publikováno v:
Genes, Vol 11, Iss 4, p 351 (2020)
Extracellular vesicles (EVs) are a class of naturally occurring secreted cellular bodies that are involved in long distance cell-to-cell communication. Proteins, lipids, mRNA, and miRNA can be packaged into these vesicles and released from the cell.
Externí odkaz:
https://doaj.org/article/a8cf445a64a344b69d3d82eeb63a0600
Publikováno v:
Genes, Vol 9, Iss 11, p 538 (2018)
Cystic fibrosis (CF) is an autosomal recessive disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that encodes a cAMP-regulated anion channel. Although CF is a multi-organ system disease, most people wi
Externí odkaz:
https://doaj.org/article/702cd35a00ed457fb509072cf112cabf
Autor:
Tayyab Rehman, Philip H. Karp, Andrew L. Thurman, Steven E. Mather, Akansha Jain, Ashley L. Cooney, Patrick L. Sinn, Alejandro A. Pezzulo, Michael E. Duffey, Michael J. Welsh
Publikováno v:
American Journal of Respiratory Cell and Molecular Biology. 67:491-502
In cystic fibrosis (CF), reduced HCOsub3/subsup-/supsecretion acidifies the airway surface liquid (ASL), and the acidic pH disrupts host defenses. Thus, understanding the control of ASL pH (pHsubASL/sub) in CF may help identify novel targets and faci
Autor:
Ashley L. Cooney, Paul B. McCray, Soumba Traore, Patrick L. Sinn, Sateesh Krishnamurthy, Christian M Brommel, Katarina Kulhankova, Gregory A. Newby, David R. Liu
Publikováno v:
Nucleic Acids Research
Mutations in the CFTR gene that lead to premature stop codons or splicing defects cause cystic fibrosis (CF) and are not amenable to treatment by small-molecule modulators. Here, we investigate the use of adenine base editor (ABE) ribonucleoproteins
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 25, Iss, Pp 293-301 (2021)
Molecular Therapy. Nucleic Acids
Molecular Therapy. Nucleic Acids
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
Autor:
Qian Dong, Ashley L. Cooney, Stefano Rivella, Paul B. McCray, Christoph O. Randak, Patrick L. Sinn, Laura I. Marquez Loza
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 21, Iss, Pp 94-106 (2021)
Molecular Therapy. Methods & Clinical Development
Molecular Therapy. Methods & Clinical Development
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