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Items: 1 to 20 of 107

1.

Systemic AAV9 gene therapy using the synapsin I promoter rescues a mouse model of neuronopathic Gaucher disease but with limited cross-correction potential to astrocytes.

Massaro G, Hughes MP, Whaler SM, Wallom KL, Priestman DA, Platt FM, Waddington SN, Rahim AA.

Hum Mol Genet. 2020 Jan 10. pii: ddz317. doi: 10.1093/hmg/ddz317. [Epub ahead of print]

PMID:
31919491
2.

Publisher Correction: In Utero Gene Therapy (IUGT) Using GLOBE Lentiviral Vector Phenotypically Corrects the Heterozygous Humanised Mouse Model and Its Progress Can Be Monitored Using MRI Techniques.

Shangaris P, Loukogeorgakis SP, Subramaniam S, Flouri C, Jackson LH, Wang W, Blundell MP, Liu S, Eaton S, Bakhamis N, Ramachandra DL, Maghsoudlou P, Urbani L, Waddington SN, Eddaoudi A, Archer J, Antoniou MN, Stuckey DJ, Schmidt M, Thrasher AJ, Ryan TM, De Coppi P, David AL.

Sci Rep. 2019 Dec 24;9(1):20214. doi: 10.1038/s41598-019-55754-y.

3.

Impaired folate one-carbon metabolism causes formate-preventable hydrocephalus in glycine decarboxylase-deficient mice.

Santos C, Pai YJ, Mahmood MR, Leung KY, Savery D, Waddington SN, Copp AJ, Greene N.

J Clin Invest. 2019 Dec 3. pii: 132360. doi: 10.1172/JCI132360. [Epub ahead of print]

4.

Continual Conscious Bioluminescent Imaging in Freely Moving Mice.

Diaz JA, Geard A, FitzPatrick LM, Delhove JMKM, Buckley SMK, Waddington SN, McKay TR, Karda R.

Methods Mol Biol. 2020;2081:161-175. doi: 10.1007/978-1-4939-9940-8_11.

PMID:
31721124
5.

Urea Cycle Related Amino Acids Measured in Dried Bloodspots Enable Long-Term In Vivo Monitoring and Therapeutic Adjustment.

Baruteau J, Khalil Y, Grunewald S, Zancolli M, Chakrapani A, Cleary M, Davison J, Footitt E, Waddington SN, Gissen P, Mills P.

Metabolites. 2019 Nov 12;9(11). pii: E275. doi: 10.3390/metabo9110275.

6.

Impaired cellular bioenergetics caused by GBA1 depletion sensitizes neurons to calcium overload.

Plotegher N, Perocheau D, Ferrazza R, Massaro G, Bhosale G, Zambon F, Rahim AA, Guella G, Waddington SN, Szabadkai G, Duchen MR.

Cell Death Differ. 2019 Nov 4. doi: 10.1038/s41418-019-0442-2. [Epub ahead of print]

PMID:
31685979
7.

In Utero Gene Therapy (IUGT) Using GLOBE Lentiviral Vector Phenotypically Corrects the Heterozygous Humanised Mouse Model and Its Progress Can Be Monitored Using MRI Techniques.

Shangaris P, Loukogeorgakis SP, Subramaniam S, Flouri C, Jackson LH, Wang W, Blundell MP, Liu S, Eaton S, Bakhamis N, Ramachandra DL, Maghsoudlou P, Urbani L, Waddington SN, Eddaoudi A, Archer J, Antoniou MN, Stuckey DJ, Schmidt M, Thrasher AJ, Ryan TM, De Coppi P, David AL.

Sci Rep. 2019 Aug 12;9(1):11592. doi: 10.1038/s41598-019-48078-4. Erratum in: Sci Rep. 2019 Dec 24;9(1):20214.

8.

In Utero Gene Therapy Consensus Statement from the IFeTIS.

Almeida-Porada G, Waddington SN, Chan JKY, Peranteau WH, MacKenzie T, Porada CD.

Mol Ther. 2019 Apr 10;27(4):705-707. doi: 10.1016/j.ymthe.2019.02.015. Epub 2019 Mar 2. No abstract available.

PMID:
30837116
9.

Fetal gene therapy for neurodegenerative lysosomal storage diseases.

Baruteau J, Waddington SN.

J Inherit Metab Dis. 2019 May;42(3):391-393. doi: 10.1002/jimd.12018. Epub 2019 Feb 4. No abstract available.

PMID:
30715735
10.

Production of lentiviral vectors using novel, enzymatically produced, linear DNA.

Karda R, Counsell JR, Karbowniczek K, Caproni LJ, Tite JP, Waddington SN.

Gene Ther. 2019 Apr;26(3-4):86-92. doi: 10.1038/s41434-018-0056-1. Epub 2019 Jan 14.

11.

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.

FASEB J. 2019 Mar;33(3):3954-3967. doi: 10.1096/fj.201801391R. Epub 2018 Dec 5.

12.

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.

Nat Commun. 2018 Aug 29;9(1):3505. doi: 10.1038/s41467-018-05972-1.

13.

Foamy Virus Vectors Transduce Visceral Organs and Hippocampal Structures following In Vivo Delivery to Neonatal Mice.

Counsell JR, Karda R, Diaz JA, Carey L, Wiktorowicz T, Buckley SMK, Ameri S, Ng J, Baruteau J, Almeida F, de Silva R, Simone R, Lugarà E, Lignani G, Lindemann D, Rethwilm A, Rahim AA, Waddington SN, Howe SJ.

Mol Ther Nucleic Acids. 2018 Sep 7;12:626-634. doi: 10.1016/j.omtn.2018.07.006. Epub 2018 Aug 3.

14.

Ascending Vaginal Infection Using Bioluminescent Bacteria Evokes Intrauterine Inflammation, Preterm Birth, and Neonatal Brain Injury in Pregnant Mice.

Suff N, Karda R, Diaz JA, Ng J, Baruteau J, Perocheau D, Tangney M, Taylor PW, Peebles D, Buckley SMK, Waddington SN.

Am J Pathol. 2018 Oct;188(10):2164-2176. doi: 10.1016/j.ajpath.2018.06.016. Epub 2018 Jul 21.

15.

Age-Related Seroprevalence of Antibodies Against AAV-LK03 in a UK Population Cohort.

Perocheau DP, Cunningham S, Lee J, Antinao Diaz J, Waddington SN, Gilmour K, Eaglestone S, Lisowski L, Thrasher AJ, Alexander IE, Gissen P, Baruteau J.

Hum Gene Ther. 2019 Jan;30(1):79-87. doi: 10.1089/hum.2018.098. Epub 2018 Oct 19.

16.

Fetal gene therapy for neurodegenerative disease of infants.

Massaro G, Mattar CNZ, Wong AMS, Sirka E, Buckley SMK, Herbert BR, Karlsson S, Perocheau DP, Burke D, Heales S, Richard-Londt A, Brandner S, Huebecker M, Priestman DA, Platt FM, Mills K, Biswas A, Cooper JD, Chan JKY, Cheng SH, Waddington SN, Rahim AA.

Nat Med. 2018 Sep;24(9):1317-1323. doi: 10.1038/s41591-018-0106-7. Epub 2018 Jul 16.

17.

AAV9 intracerebroventricular gene therapy improves lifespan, locomotor function and pathology in a mouse model of Niemann-Pick type C1 disease.

Hughes MP, Smith DA, Morris L, Fletcher C, Colaco A, Huebecker M, Tordo J, Palomar N, Massaro G, Henckaerts E, Waddington SN, Platt FM, Rahim AA.

Hum Mol Genet. 2018 Sep 1;27(17):3079-3098. doi: 10.1093/hmg/ddy212.

18.

A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency.

Tordo J, O'Leary C, Antunes ASLM, Palomar N, Aldrin-Kirk P, Basche M, Bennett A, D'Souza Z, Gleitz H, Godwin A, Holley RJ, Parker H, Liao AY, Rouse P, Youshani AS, Dridi L, Martins C, Levade T, Stacey KB, Davis DM, Dyer A, Clément N, Björklund T, Ali RR, Agbandje-McKenna M, Rahim AA, Pshezhetsky A, Waddington SN, Linden RM, Bigger BW, Henckaerts E.

Brain. 2018 Jul 1;141(7):2014-2031. doi: 10.1093/brain/awy126.

19.

NF-κB Activity Initiates Human ESC-Derived Neural Progenitor Cell Differentiation by Inducing a Metabolic Maturation Program.

FitzPatrick LM, Hawkins KE, Delhove JMKM, Fernandez E, Soldati C, Bullen LF, Nohturfft A, Waddington SN, Medina DL, Bolaños JP, McKay TR.

Stem Cell Reports. 2018 Jun 5;10(6):1766-1781. doi: 10.1016/j.stemcr.2018.03.015. Epub 2018 Apr 19.

20.

A comparison of intrauterine hemopoietic cell transplantation and lentiviral gene transfer for the correction of severe β-thalassemia in a HbbTh3/+ murine model.

Dighe NM, Tan KW, Tan LG, Shaw SSW, Buckley SMK, Sandikin D, Johana N, Tan YW, Biswas A, Choolani M, Waddington SN, Antoniou MN, Chan JKY, Mattar CNZ.

Exp Hematol. 2018 Jun;62:45-55. doi: 10.1016/j.exphem.2018.03.006. Epub 2018 Mar 29.

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