P01 HD032652/HD/NICHD NIH HHS/United States[Grant Number] - Search Results

Year	Number of Results
1998	1
2004	1
2005	3
2006	4
2007	6
2008	3
2009	1
2010	2
2011	6
2012	4
2013	5
2014	10
2015	5
2016	3
2017	5
2018	2
2019	2
2020	1
2024	0

1

Prevalence, incidence and carrier frequency of 5q-linked spinal muscular atrophy - a literature review.

Verhaart IEC, Robertson A, Wilson IJ, Aartsma-Rus A, Cameron S, Jones CC, Cook SF, Lochmüller H. Verhaart IEC, et al. Orphanet J Rare Dis. 2017 Jul 4;12(1):124. doi: 10.1186/s13023-017-0671-8. Orphanet J Rare Dis. 2017. PMID: 28676062 Free PMC article. Review.

2

Precision editing of large animal genomes.

Tan WS, Carlson DF, Walton MW, Fahrenkrug SC, Hackett PB. Tan WS, et al. Adv Genet. 2012;80:37-97. doi: 10.1016/B978-0-12-404742-6.00002-8. Adv Genet. 2012. PMID: 23084873 Free PMC article. Review.

3

Gene therapy for neurologic manifestations of mucopolysaccharidoses.

Wolf DA, Banerjee S, Hackett PB, Whitley CB, McIvor RS, Low WC. Wolf DA, et al. Expert Opin Drug Deliv. 2015 Feb;12(2):283-96. doi: 10.1517/17425247.2015.966682. Epub 2014 Dec 16. Expert Opin Drug Deliv. 2015. PMID: 25510418 Free PMC article. Review.

4

The Sleeping Beauty transposon system: a non-viral vector for gene therapy.

Aronovich EL, McIvor RS, Hackett PB. Aronovich EL, et al. Hum Mol Genet. 2011 Apr 15;20(R1):R14-20. doi: 10.1093/hmg/ddr140. Epub 2011 Apr 1. Hum Mol Genet. 2011. PMID: 21459777 Free PMC article. Review.

5

A transposon and transposase system for human application.

Hackett PB, Largaespada DA, Cooper LJ. Hackett PB, et al. Mol Ther. 2010 Apr;18(4):674-83. doi: 10.1038/mt.2010.2. Epub 2010 Jan 26. Mol Ther. 2010. PMID: 20104209 Free PMC article. Review.

6

Efficacy and safety of Sleeping Beauty transposon-mediated gene transfer in preclinical animal studies.

Hackett PB Jr, Aronovich EL, Hunter D, Urness M, Bell JB, Kass SJ, Cooper LJ, McIvor S. Hackett PB Jr, et al. Curr Gene Ther. 2011 Oct;11(5):341-9. doi: 10.2174/156652311797415827. Curr Gene Ther. 2011. PMID: 21888621 Free PMC article. Review.

7

Evaluating risks of insertional mutagenesis by DNA transposons in gene therapy.

Hackett PB, Largaespada DA, Switzer KC, Cooper LJ. Hackett PB, et al. Transl Res. 2013 Apr;161(4):265-83. doi: 10.1016/j.trsl.2012.12.005. Epub 2013 Jan 10. Transl Res. 2013. PMID: 23313630 Free PMC article. Review.

8

Lysosomal storage disease: gene therapy on both sides of the blood-brain barrier.

Aronovich EL, Hackett PB. Aronovich EL, et al. Mol Genet Metab. 2015 Feb;114(2):83-93. doi: 10.1016/j.ymgme.2014.09.011. Epub 2014 Oct 7. Mol Genet Metab. 2015. PMID: 25410058 Free PMC article. Review.

9

Genotype-phenotype correlation of gangliosidosis mutations using in silico tools and homology modeling.

Ou L, Kim S, Whitley CB, Jarnes-Utz JR. Ou L, et al. Mol Genet Metab Rep. 2019 Jul 17;20:100495. doi: 10.1016/j.ymgmr.2019.100495. eCollection 2019 Sep. Mol Genet Metab Rep. 2019. PMID: 31367523 Free PMC article.

10

Sleeping beauty transposon-mediated gene therapy for prolonged expression.

Hackett PB, Ekker SC, Largaespada DA, McIvor RS. Hackett PB, et al. Adv Genet. 2005;54:189-232. doi: 10.1016/S0065-2660(05)54009-4. Adv Genet. 2005. PMID: 16096013 Review.

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