New Directions in Pulmonary Gene Therapy.

作者: Amber Vu , Paul B. McCray

DOI: 10.1089/HUM.2020.166

关键词:

摘要: … dual Cas12a targeting of these loxP sequences results in localized GFP fluorescence. This study shows the direct application of Cas12a … These rAAV/HBoV or GBoV chimeras effectively …

参考文章(246)
Ziying Yan, Xingshen Sun, Zehua Feng, Guiying Li, John T. Fisher, Zoe A. Stewart, John F. Engelhardt, Optimization of Recombinant Adeno-Associated Virus-Mediated Expression for Large Transgenes, Using a Synthetic Promoter and Tandem Array Enhancers Human Gene Therapy. ,vol. 26, pp. 334- 346 ,(2015) , 10.1089/HUM.2015.001
Maria J. Chiuchiolo, Stephen M. Kaminsky, Dolan Sondhi, Denesy Mancenido, Charleen Hollmann, Ronald G. Crystal, Phase I/II study of intrapleural administration of a serotype rh.10 replication-deficient adeno-associated virus gene transfer vector expressing the human α1-antitrypsin cDNA to individuals with α1-antitrypsin deficiency. Human gene therapy. Clinical development. ,vol. 25, pp. 112- 133 ,(2014) , 10.1089/HUMC.2014.2513
Gary P. Kobinger, Daniel J. Weiner, Qian-Chun Yu, James M. Wilson, Filovirus-pseudotyped lentiviral vector can efficiently and stably transduce airway epithelia in vivo. Nature Biotechnology. ,vol. 19, pp. 225- 230 ,(2001) , 10.1038/85664
S. E. Raper, J. M. Wilson, M. Yudkoff, M. B. Robinson, X. Ye, M. L. Batshaw, Developing adenoviral-mediated in vivo gene therapy for ornithine transcarbamylase deficiency Journal of Inherited Metabolic Disease. ,vol. 21, pp. 119- 137 ,(1998) , 10.1023/A:1005369926784
Claire E Wainwright, J Stuart Elborn, Bonnie W Ramsey, Gautham Marigowda, Xiaohong Huang, Marco Cipolli, Carla Colombo, Jane C Davies, Kris De Boeck, Patrick A Flume, Michael W Konstan, Susanna A McColley, Karen McCoy, Edward F McKone, Anne Munck, Felix Ratjen, Steven M Rowe, David Waltz, Michael P Boyle, None, Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR The New England Journal of Medicine. ,vol. 373, pp. 220- 231 ,(2015) , 10.1056/NEJMOA1409547
Amy L Firth, Tushar Menon, Gregory S Parker, Susan J Qualls, Benjamin M Lewis, Eugene Ke, Carl T Dargitz, Rebecca Wright, Ajai Khanna, Fred H Gage, Inder M Verma, None, Functional Gene Correction for Cystic Fibrosis in Lung Epithelial Cells Generated from Patient iPSCs Cell Reports. ,vol. 12, pp. 1385- 1390 ,(2015) , 10.1016/J.CELREP.2015.07.062
Dong-Jiunn Jeffery Truong, Karin Kühner, Ralf Kühn, Stanislas Werfel, Stefan Engelhardt, Wolfgang Wurst, Oskar Ortiz, Development of an intein-mediated split–Cas9 system for gene therapy Nucleic Acids Research. ,vol. 43, pp. 6450- 6458 ,(2015) , 10.1093/NAR/GKV601
Azita J Mahiny, Alexander Dewerth, Lauren E Mays, Mohammed Alkhaled, Benedikt Mothes, Emad Malaeksefat, Brigitta Loretz, Jennifer Rottenberger, Darina M Brosch, Philipp Reautschnig, Pacharapan Surapolchai, Franziska Zeyer, Andrea Schams, Melanie Carevic, Martina Bakele, Matthias Griese, Matthias Schwab, Bernd Nürnberg, Sandra Beer-Hammer, Rupert Handgretinger, Dominik Hartl, Claus-Michael Lehr, Michael S D Kormann, In vivo genome editing using nuclease-encoding mRNA corrects SP-B deficiency Nature Biotechnology. ,vol. 33, pp. 584- 586 ,(2015) , 10.1038/NBT.3241
Eric W F W Alton, David K Armstrong, Deborah Ashby, Katie J Bayfield, Diana Bilton, Emily V Bloomfield, A Christopher Boyd, June Brand, Ruaridh Buchan, Roberto Calcedo, Paula Carvelli, Mario Chan, Seng H Cheng, D David S Collie, Steve Cunningham, Heather E Davidson, Gwyneth Davies, Jane C Davies, Lee A Davies, Maria H Dewar, Ann Doherty, Jackie Donovan, Natalie S Dwyer, Hala I Elgmati, Rosanna F Featherstone, Jemyr Gavino, Sabrina Gea-Sorli, Duncan M Geddes, James S R Gibson, Deborah R Gill, Andrew P Greening, Uta Griesenbach, David M Hansell, Katharine Harman, Tracy E Higgins, Samantha L Hodges, Stephen C Hyde, Laura Hyndman, J Alastair Innes, Joseph Jacob, Nancy Jones, Brian F Keogh, Maria P Limberis, Paul Lloyd-Evans, Alan W Maclean, Michelle C Manvell, Dominique McCormick, Michael McGovern, Gerry McLachlan, Cuixiang Meng, M Angeles Montero, Hazel Milligan, Laura J Moyce, Gordon D Murray, Andrew G Nicholson, Tina Osadolor, Javier Parra-Leiton, David J Porteous, Ian A Pringle, Emma K Punch, Kamila M Pytel, Alexandra L Quittner, Gina Rivellini, Clare J Saunders, Ronald K Scheule, Sarah Sheard, Nicholas J Simmonds, Keith Smith, Stephen N Smith, Najwa Soussi, Samia Soussi, Emma J Spearing, Barbara J Stevenson, Stephanie G Sumner-Jones, Minna Turkkila, Rosa P Ureta, Michael D Waller, Marguerite Y Wasowicz, James M Wilson, Paul Wolstenholme-Hogg, Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial The Lancet Respiratory Medicine. ,vol. 3, pp. 684- 691 ,(2015) , 10.1016/S2213-2600(15)00245-3