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Re-structuring lentiviral vectors to express genomic RNA via cap-dependent translation.
Counsell JR, De Brabandere G, Karda R, Moore M, Greco A, Bray A, Diaz JA, Perocheau DP, Mock U, Waddington SN. Counsell JR, et al. Among authors: waddington sn. Mol Ther Methods Clin Dev. 2020 Dec 15;20:357-365. doi: 10.1016/j.omtm.2020.12.005. eCollection 2021 Mar 12. Mol Ther Methods Clin Dev. 2020. PMID: 33553484 Free PMC article.
Normally, this RNA genome is reverse transcribed into a double-stranded DNA provirus that integrates into the cell genome, providing permanent gene transfer and long-term expression. Integration-deficient LV vectors have been developed to reduce the frequency of genomic in …
Normally, this RNA genome is reverse transcribed into a double-stranded DNA provirus that integrates into the cell genome, providing permane …
Continual Conscious Bioluminescent Imaging in Freely Moving Mice.
Diaz JA, Geard A, FitzPatrick LM, Delhove JMKM, Buckley SMK, Waddington SN, McKay TR, Karda R. Diaz JA, et al. Among authors: waddington sn. Methods Mol Biol. 2020;2081:161-175. doi: 10.1007/978-1-4939-9940-8_11. Methods Mol Biol. 2020. PMID: 31721124
Here we describe a novel technology whereby we can generate somatotransgenic rodents with the use of a viral vector carrying a luciferase transgene. We are able to achieve long term luciferase expression by a single injection of lentiviral or adeno-associated virus vectors …
Here we describe a novel technology whereby we can generate somatotransgenic rodents with the use of a viral vector carrying a luciferase tr …
Production of lentiviral vectors using novel, enzymatically produced, linear DNA.
Karda R, Counsell JR, Karbowniczek K, Caproni LJ, Tite JP, Waddington SN. Karda R, et al. Among authors: waddington sn. Gene Ther. 2019 Apr;26(3-4):86-92. doi: 10.1038/s41434-018-0056-1. Epub 2019 Jan 14. Gene Ther. 2019. PMID: 30643205 Free PMC article.
Luciferase expression was quantified in conscious mice continually by whole-body bioluminescent imaging. We observed long-term luciferase expression using dbDNA-derived vectors, which was comparable to plasmid-derived lentivirus vectors. ...
Luciferase expression was quantified in conscious mice continually by whole-body bioluminescent imaging. We observed long-term lucife …
Therapeutic expression of human clotting factors IX and X following adeno-associated viral vector-mediated intrauterine gene transfer in early-gestation fetal macaques.
Chan JKY, Gil-Farina I, Johana N, Rosales C, Tan YW, Ceiler J, Mcintosh J, Ogden B, Waddington SN, Schmidt M, Biswas A, Choolani M, Nathwani AC, Mattar CNZ. Chan JKY, et al. Among authors: waddington sn. FASEB J. 2019 Mar;33(3):3954-3967. doi: 10.1096/fj.201801391R. Epub 2018 Dec 5. FASEB J. 2019. PMID: 30517034 Free PMC article.
The initial cohort received 1 10(12) vector genomes (vgs) of AAV5-hFIX ( n = 5; 0.45 10(13) vg/kg birth weight), resulting in 3.0% hFIX at birth and 0.6-6.8% over 19-51 mo. ...AAV5-hFX animals ( n = 3; 3.57 10(13) vg/kg) expressed <1% at birth and 9.4-27.9 …
The initial cohort received 1 10(12) vector genomes (vgs) of AAV5-hFIX ( n = 5; 0.45 10(13) vg/kg birth weight), resulting in 3.0% hF …
Argininosuccinic aciduria fosters neuronal nitrosative stress reversed by Asl gene transfer.
Baruteau J, Perocheau DP, Hanley J, Lorvellec M, Rocha-Ferreira E, Karda R, Ng J, Suff N, Diaz JA, Rahim AA, Hughes MP, Banushi B, Prunty H, Hristova M, Ridout DA, Virasami A, Heales S, Howe SJ, Buckley SMK, Mills PB, Gissen P, Waddington SN. Baruteau J, et al. Among authors: waddington sn. Nat Commun. 2018 Aug 29;9(1):3505. doi: 10.1038/s41467-018-05972-1. Nat Commun. 2018. PMID: 30158522 Free PMC article.
Intravenous injection of AAV8 vector into adult or neonatal ASL-deficient mice demonstrates long-term correction of the hepatic urea cycle and the cerebral citrulline-NO cycle, respectively. ...
Intravenous injection of AAV8 vector into adult or neonatal ASL-deficient mice demonstrates long-term correction of the hepatic urea …
High-efficiency transduction of spinal cord motor neurons by intrauterine delivery of integration-deficient lentiviral vectors.
Ahmed SG, Waddington SN, Boza-Morán MG, Yáñez-Muñoz RJ. Ahmed SG, et al. Among authors: waddington sn. J Control Release. 2018 Mar 10;273:99-107. doi: 10.1016/j.jconrel.2017.12.029. Epub 2017 Dec 28. J Control Release. 2018. PMID: 29289570 Free PMC article.
Integration-deficient lentiviral vectors (IDLVs) are promising gene delivery tools that retain the high transduction efficiency of standard lentiviral vectors, yet fail to integrate as proviruses and are instead converted into episomal circles. These episomes are metabolically st …
Integration-deficient lentiviral vectors (IDLVs) are promising gene delivery tools that retain the high transduction efficiency of standard …
Progesterone, the maternal immune system and the onset of parturition in the mouse.
Edey LF, Georgiou H, O'Dea KP, Mesiano S, Herbert BR, Lei K, Hua R, Markovic D, Waddington SN, MacIntyre D, Bennett P, Takata M, Johnson MR. Edey LF, et al. Among authors: waddington sn. Biol Reprod. 2018 Mar 1;98(3):376-395. doi: 10.1093/biolre/iox146. Biol Reprod. 2018. PMID: 29145579 Free article.
The role of progesterone (P4) in the regulation of the local (uterine) and systemic innate immune system, myometrial expression of connexin 43 (Cx-43) and cyclooxygenase 2 (COX-2), and the onset of parturition was examined in (i) naive mice delivering at term; (ii) E16 mic …
The role of progesterone (P4) in the regulation of the local (uterine) and systemic innate immune system, myometrial expression of connexin …
Enhancement of mouse hematopoietic stem/progenitor cell function via transient gene delivery using integration-deficient lentiviral vectors.
Alonso-Ferrero ME, van Til NP, Bartolovic K, Mata MF, Wagemaker G, Moulding D, Williams DA, Kinnon C, Waddington SN, Milsom MD, Howe SJ. Alonso-Ferrero ME, et al. Among authors: waddington sn. Exp Hematol. 2018 Jan;57:21-29. doi: 10.1016/j.exphem.2017.09.003. Epub 2017 Sep 11. Exp Hematol. 2018. PMID: 28911908 Free PMC article.
This property can be harnessed to express transgenes transiently to manipulate cell biology. Here, we demonstrate the utility of short-term gene expression to improve functional potency of hematopoietic stem and progenitor cells (HSPCs) during transplantation by delivering …
This property can be harnessed to express transgenes transiently to manipulate cell biology. Here, we demonstrate the utility of short-te …
Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects.
Baruteau J, Waddington SN, Alexander IE, Gissen P. Baruteau J, et al. Among authors: waddington sn. J Inherit Metab Dis. 2017 Jul;40(4):497-517. doi: 10.1007/s10545-017-0053-3. Epub 2017 May 31. J Inherit Metab Dis. 2017. PMID: 28567541 Free PMC article. Review.
Gene therapy is likely to become an option for routine care of a subset of severe inherited genetic/metabolic liver diseases in the relatively near term. In this review, we aim to summarise the milestones in the development of gene therapy, present the different vector too …
Gene therapy is likely to become an option for routine care of a subset of severe inherited genetic/metabolic liver diseases in the relative …
37 results