Matches in SemOpenAlex for { <https://semopenalex.org/work/W2994805025> ?p ?o ?g. }
- W2994805025 endingPage "171.e4" @default.
- W2994805025 startingPage "161" @default.
- W2994805025 abstract "Cystic fibrosis (CF) is a monogenic disorder caused by mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. Mortality in CF patients is mostly due to respiratory sequelae. Challenges with gene delivery have limited attempts to treat CF using in vivo gene therapy, and low correction levels have hindered ex vivo gene therapy efforts. We have used Cas9 and adeno-associated virus 6 to correct the ΔF508 mutation in readily accessible upper-airway basal stem cells (UABCs) obtained from CF patients. On average, we achieved 30%-50% allelic correction in UABCs and bronchial epithelial cells (HBECs) from 10 CF patients and observed 20%-50% CFTR function relative to non-CF controls in differentiated epithelia. Furthermore, we successfully embedded the corrected UABCs on an FDA-approved porcine small intestinal submucosal membrane (pSIS), and they retained differentiation capacity. This study supports further development of genetically corrected autologous airway stem cell transplant as a treatment for CF." @default.
- W2994805025 created "2019-12-26" @default.
- W2994805025 creator A5000912039 @default.
- W2994805025 creator A5007644395 @default.
- W2994805025 creator A5007678351 @default.
- W2994805025 creator A5012270723 @default.
- W2994805025 creator A5019640686 @default.
- W2994805025 creator A5022593117 @default.
- W2994805025 creator A5040157182 @default.
- W2994805025 creator A5044985085 @default.
- W2994805025 creator A5047595168 @default.
- W2994805025 creator A5047622778 @default.
- W2994805025 creator A5051621625 @default.
- W2994805025 creator A5051925609 @default.
- W2994805025 creator A5052721402 @default.
- W2994805025 creator A5054698030 @default.
- W2994805025 creator A5056326884 @default.
- W2994805025 creator A5056685479 @default.
- W2994805025 creator A5058576720 @default.
- W2994805025 creator A5071404060 @default.
- W2994805025 creator A5071885288 @default.
- W2994805025 creator A5076054268 @default.
- W2994805025 creator A5079842773 @default.
- W2994805025 creator A5079921861 @default.
- W2994805025 creator A5081026957 @default.
- W2994805025 creator A5088028354 @default.
- W2994805025 date "2020-02-01" @default.
- W2994805025 modified "2023-10-16" @default.
- W2994805025 title "High-Efficiency, Selection-free Gene Repair in Airway Stem Cells from Cystic Fibrosis Patients Rescues CFTR Function in Differentiated Epithelia" @default.
- W2994805025 cites W1506907398 @default.
- W2994805025 cites W1589654967 @default.
- W2994805025 cites W1617270423 @default.
- W2994805025 cites W1922064584 @default.
- W2994805025 cites W1978991565 @default.
- W2994805025 cites W1986316308 @default.
- W2994805025 cites W1990991203 @default.
- W2994805025 cites W2007914388 @default.
- W2994805025 cites W2018456465 @default.
- W2994805025 cites W2035131077 @default.
- W2994805025 cites W2036176224 @default.
- W2994805025 cites W2048303863 @default.
- W2994805025 cites W2049814146 @default.
- W2994805025 cites W2061572587 @default.
- W2994805025 cites W2061752094 @default.
- W2994805025 cites W2067432020 @default.
- W2994805025 cites W2071407356 @default.
- W2994805025 cites W2073843991 @default.
- W2994805025 cites W2076138292 @default.
- W2994805025 cites W2082968284 @default.
- W2994805025 cites W2088572648 @default.
- W2994805025 cites W2091140839 @default.
- W2994805025 cites W2092505971 @default.
- W2994805025 cites W2111856827 @default.
- W2994805025 cites W2112250552 @default.
- W2994805025 cites W2128758977 @default.
- W2994805025 cites W2150297577 @default.
- W2994805025 cites W2159582900 @default.
- W2994805025 cites W2177438840 @default.
- W2994805025 cites W2209402024 @default.
- W2994805025 cites W2317788533 @default.
- W2994805025 cites W2344364216 @default.
- W2994805025 cites W2514072306 @default.
- W2994805025 cites W2547418311 @default.
- W2994805025 cites W2574405809 @default.
- W2994805025 cites W2771225072 @default.
- W2994805025 cites W2784423079 @default.
- W2994805025 cites W2802473049 @default.
- W2994805025 cites W2806797030 @default.
- W2994805025 cites W2810403368 @default.
- W2994805025 cites W2897626245 @default.
- W2994805025 cites W2942076130 @default.
- W2994805025 cites W2949380898 @default.
- W2994805025 cites W2952346338 @default.
- W2994805025 cites W4206217612 @default.
- W2994805025 cites W4245053291 @default.
- W2994805025 cites W909607896 @default.
- W2994805025 doi "https://doi.org/10.1016/j.stem.2019.11.002" @default.
- W2994805025 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/31839569" @default.
- W2994805025 hasPublicationYear "2020" @default.
- W2994805025 type Work @default.
- W2994805025 sameAs 2994805025 @default.
- W2994805025 citedByCount "81" @default.
- W2994805025 countsByYear W29948050252020 @default.
- W2994805025 countsByYear W29948050252021 @default.
- W2994805025 countsByYear W29948050252022 @default.
- W2994805025 countsByYear W29948050252023 @default.
- W2994805025 crossrefType "journal-article" @default.
- W2994805025 hasAuthorship W2994805025A5000912039 @default.
- W2994805025 hasAuthorship W2994805025A5007644395 @default.
- W2994805025 hasAuthorship W2994805025A5007678351 @default.
- W2994805025 hasAuthorship W2994805025A5012270723 @default.
- W2994805025 hasAuthorship W2994805025A5019640686 @default.
- W2994805025 hasAuthorship W2994805025A5022593117 @default.
- W2994805025 hasAuthorship W2994805025A5040157182 @default.
- W2994805025 hasAuthorship W2994805025A5044985085 @default.
- W2994805025 hasAuthorship W2994805025A5047595168 @default.
- W2994805025 hasAuthorship W2994805025A5047622778 @default.
- W2994805025 hasAuthorship W2994805025A5051621625 @default.