Batshaw ML - Search Results

1

Vector sequences are not detected in tumor tissue from research subjects with ornithine transcarbamylase deficiency who previously received adenovirus gene transfer.

Zhong L, Li S, Li M, Xie J, Zhang Y, Lee B, Batshaw ML, Wilson JM, Gao G. Zhong L, et al. Among authors: batshaw ml. Hum Gene Ther. 2013 Sep;24(9):814-9. doi: 10.1089/hum.2013.118. Hum Gene Ther. 2013. PMID: 24010702 Free PMC article.

2

A pilot study of in vivo liver-directed gene transfer with an adenoviral vector in partial ornithine transcarbamylase deficiency.

Raper SE, Yudkoff M, Chirmule N, Gao GP, Nunes F, Haskal ZJ, Furth EE, Propert KJ, Robinson MB, Magosin S, Simoes H, Speicher L, Hughes J, Tazelaar J, Wivel NA, Wilson JM, Batshaw ML. Raper SE, et al. Among authors: batshaw ml. Hum Gene Ther. 2002 Jan 1;13(1):163-75. doi: 10.1089/10430340152712719. Hum Gene Ther. 2002. PMID: 11779420

3

Long-term correction of ammonia metabolism and prolonged survival in ornithine transcarbamylase-deficient mice following liver-directed treatment with adeno-associated viral vectors.

Moscioni D, Morizono H, McCarter RJ, Stern A, Cabrera-Luque J, Hoang A, Sanmiguel J, Wu D, Bell P, Gao GP, Raper SE, Wilson JM, Batshaw ML. Moscioni D, et al. Among authors: batshaw ml. Mol Ther. 2006 Jul;14(1):25-33. doi: 10.1016/j.ymthe.2006.03.009. Epub 2006 May 3. Mol Ther. 2006. PMID: 16677864 Free article.

4

Analysis of tumors arising in male B6C3F1 mice with and without AAV vector delivery to liver.

Bell P, Moscioni AD, McCarter RJ, Wu D, Gao G, Hoang A, Sanmiguel JC, Sun X, Wivel NA, Raper SE, Furth EE, Batshaw ML, Wilson JM. Bell P, et al. Among authors: batshaw ml. Mol Ther. 2006 Jul;14(1):34-44. doi: 10.1016/j.ymthe.2006.03.008. Epub 2006 May 6. Mol Ther. 2006. PMID: 16682254 Free article.

5

Hepatocellular carcinoma in a research subject with ornithine transcarbamylase deficiency.

Wilson JM, Shchelochkov OA, Gallagher RC, Batshaw ML. Wilson JM, et al. Among authors: batshaw ml. Mol Genet Metab. 2012 Feb;105(2):263-5. doi: 10.1016/j.ymgme.2011.10.016. Epub 2011 Nov 7. Mol Genet Metab. 2012. PMID: 22129577 Free PMC article.

6

Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer.

Raper SE, Chirmule N, Lee FS, Wivel NA, Bagg A, Gao GP, Wilson JM, Batshaw ML. Raper SE, et al. Among authors: batshaw ml. Mol Genet Metab. 2003 Sep-Oct;80(1-2):148-58. doi: 10.1016/j.ymgme.2003.08.016. Mol Genet Metab. 2003. PMID: 14567964 Clinical Trial.

7

Systematic evaluation of AAV vectors for liver directed gene transfer in murine models.

Wang L, Wang H, Bell P, McCarter RJ, He J, Calcedo R, Vandenberghe LH, Morizono H, Batshaw ML, Wilson JM. Wang L, et al. Among authors: batshaw ml. Mol Ther. 2010 Jan;18(1):118-25. doi: 10.1038/mt.2009.246. Epub 2009 Oct 27. Mol Ther. 2010. PMID: 19861950 Free PMC article.

8

The pleiotropic effects of natural AAV infections on liver-directed gene transfer in macaques.

Wang L, Calcedo R, Wang H, Bell P, Grant R, Vandenberghe LH, Sanmiguel J, Morizono H, Batshaw ML, Wilson JM. Wang L, et al. Among authors: batshaw ml. Mol Ther. 2010 Jan;18(1):126-34. doi: 10.1038/mt.2009.245. Epub 2009 Nov 3. Mol Ther. 2010. PMID: 19888196 Free PMC article.

9

Preclinical evaluation of a clinical candidate AAV8 vector for ornithine transcarbamylase (OTC) deficiency reveals functional enzyme from each persisting vector genome.

Wang L, Morizono H, Lin J, Bell P, Jones D, McMenamin D, Yu H, Batshaw ML, Wilson JM. Wang L, et al. Among authors: batshaw ml. Mol Genet Metab. 2012 Feb;105(2):203-11. doi: 10.1016/j.ymgme.2011.10.020. Epub 2011 Nov 7. Mol Genet Metab. 2012. PMID: 22133298 Free PMC article.

10

A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice.

Yang Y, Wang L, Bell P, McMenamin D, He Z, White J, Yu H, Xu C, Morizono H, Musunuru K, Batshaw ML, Wilson JM. Yang Y, et al. Among authors: batshaw ml. Nat Biotechnol. 2016 Mar;34(3):334-8. doi: 10.1038/nbt.3469. Epub 2016 Feb 1. Nat Biotechnol. 2016. PMID: 26829317 Free PMC article.

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