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Gene Therapy Potential for Genetic Disorders of Surfactant Dysfunction.
Cooney AL, Wambach JA, Sinn PL, McCray PB Jr. Cooney AL, et al. Among authors: mccray pb jr. Front Genome Ed. 2022 Jan 14;3:785829. doi: 10.3389/fgeed.2021.785829. eCollection 2021. Front Genome Ed. 2022. PMID: 35098209 Free PMC article. Review.
While respiratory distress syndrome (RDS) is common in premature infants, severe RDS in term and late preterm infants suggests an underlying genetic etiology. ...
While respiratory distress syndrome (RDS) is common in premature infants, severe RDS in term and late preterm infants suggests an und …
Engineered amphiphilic peptides enable delivery of proteins and CRISPR-associated nucleases to airway epithelia.
Krishnamurthy S, Wohlford-Lenane C, Kandimalla S, Sartre G, Meyerholz DK, Théberge V, Hallée S, Duperré AM, Del'Guidice T, Lepetit-Stoffaes JP, Barbeau X, Guay D, McCray PB Jr. Krishnamurthy S, et al. Among authors: mccray pb jr. Nat Commun. 2019 Oct 28;10(1):4906. doi: 10.1038/s41467-019-12922-y. Nat Commun. 2019. PMID: 31659165 Free PMC article.
Instillation of shuttle peptides combined with SpCas9 or AsCas12a RNP achieves editing of loxP sites in airway epithelia of ROSA(mT/mG) mice. We observe no evidence of short-term toxicity with a widespread distribution restricted to the respiratory tract. ...
Instillation of shuttle peptides combined with SpCas9 or AsCas12a RNP achieves editing of loxP sites in airway epithelia of ROSA(mT/mG) mice …
A Novel AAV-mediated Gene Delivery System Corrects CFTR Function in Pigs.
Cooney AL, Thornell IM, Singh BK, Shah VS, Stoltz DA, McCray PB Jr, Zabner J, Sinn PL. Cooney AL, et al. Among authors: mccray pb jr. Am J Respir Cell Mol Biol. 2019 Dec;61(6):747-754. doi: 10.1165/rcmb.2019-0006OC. Am J Respir Cell Mol Biol. 2019. PMID: 31184507 Free PMC article.
Because of the packaging constraints of AAV, careful consideration was required to ensure that the vector would package and express its CFTR cDNA cargo. In this short-term study, PB/AAV-CFTR was aerosolized to the airways of CF pigs in the absence of the transposase. ...
Because of the packaging constraints of AAV, careful consideration was required to ensure that the vector would package and express its CFTR …
Lentiviral Vectors for the Treatment and Prevention of Cystic Fibrosis Lung Disease.
Marquez Loza LI, Yuen EC, McCray PB Jr. Marquez Loza LI, et al. Among authors: mccray pb jr. Genes (Basel). 2019 Mar 14;10(3):218. doi: 10.3390/genes10030218. Genes (Basel). 2019. PMID: 30875857 Free PMC article. Review.
Lentiviral vectors are well-suited for this purpose because they transduce nondividing cells, and provide long-term transgene expression. Studies in primary cultures of human CF airway epithelia and CF animal models demonstrate the long-term correction of CF phenoty …
Lentiviral vectors are well-suited for this purpose because they transduce nondividing cells, and provide long-term transgene express …
piggyBac-mediated phenotypic correction of factor VIII deficiency.
Staber JM, Pollpeter MJ, Arensdorf A, Sinn PL, Rutkowski DT, McCray PB Jr. Staber JM, et al. Among authors: mccray pb jr. Mol Ther Methods Clin Dev. 2014 Sep 10;1:14042. doi: 10.1038/mtm.2014.42. eCollection 2014. Mol Ther Methods Clin Dev. 2014. PMID: 26015980 Free PMC article.
While several vectors are under investigation, gene addition from an integrated transgene offers the possibility of long term expression. We engineered the DNA transposon-based vector, piggyBac (PB), to carry a codon-optimized B-domain deleted human FVIII cDNA. ...These da …
While several vectors are under investigation, gene addition from an integrated transgene offers the possibility of long term express …
Integrase-defective lentiviral vectors: progress and applications.
Banasik MB, McCray PB Jr. Banasik MB, et al. Among authors: mccray pb jr. Gene Ther. 2010 Feb;17(2):150-7. doi: 10.1038/gt.2009.135. Epub 2009 Oct 22. Gene Ther. 2010. PMID: 19847206 Review.
Lentiviral vectors (LVs) offer the advantages of a large packaging capacity, broad cell tropism or specific cell-type targeting through pseudotyping, and long-term expression from integrated gene cassettes. However, transgene integration carries a risk of disrupting gene e …
Lentiviral vectors (LVs) offer the advantages of a large packaging capacity, broad cell tropism or specific cell-type targeting through pseu …
Gene therapy progress and prospects: development of improved lentiviral and retroviral vectors--design, biosafety, and production.
Sinn PL, Sauter SL, McCray PB Jr. Sinn PL, et al. Among authors: mccray pb jr. Gene Ther. 2005 Jul;12(14):1089-98. doi: 10.1038/sj.gt.3302570. Gene Ther. 2005. PMID: 16003340 Review.
Replication defective vectors derived from simple retroviruses or the more complex genomes of lentiviruses continue to offer the advantages of long-term expression, cell and tissue specific tropism, and large packaging capacity for the delivery of therapeutic genes. ...
Replication defective vectors derived from simple retroviruses or the more complex genomes of lentiviruses continue to offer the advantages …
In vivo treatment of hemophilia A and mucopolysaccharidosis type VII using nonprimate lentiviral vectors.
Stein CS, Kang Y, Sauter SL, Townsend K, Staber P, Derksen TA, Martins I, Qian J, Davidson BL, McCray PB Jr. Stein CS, et al. Among authors: mccray pb jr. Mol Ther. 2001 Jun;3(6):850-6. doi: 10.1006/mthe.2001.0325. Mol Ther. 2001. PMID: 11407898 Free article.
These findings indicate that gene transfer with FIV-based lentiviral vectors can permanently introduce transgenes into a sufficient number of hepatocytes for long-term therapeutic effect and suggest potential clinical value of FIV-based lentiviral vectors for treatment of …
These findings indicate that gene transfer with FIV-based lentiviral vectors can permanently introduce transgenes into a sufficient number o …
Transient surfactant protein B deficiency in a term infant with severe respiratory failure.
Klein JM, Thompson MW, Snyder JM, George TN, Whitsett JA, Bell EF, McCray PB Jr, Nogee LM. Klein JM, et al. Among authors: mccray pb jr. J Pediatr. 1998 Feb;132(2):244-8. doi: 10.1016/s0022-3476(98)70439-1. J Pediatr. 1998. PMID: 9506635
A 38-day-old male infant with persistent pulmonary hypertension and respiratory failure since birth was found to have a complete absence of surfactant protein B (SP-B) along with an aberrant form of SP-C in his tracheal aspirate fluid, findings consistent with the diagnosi …
A 38-day-old male infant with persistent pulmonary hypertension and respiratory failure since birth was found to have a complete abse …