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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. Massaro G, et al. Among authors: mattar cnz. Nat Med. 2018 Sep;24(9):1317-1323. doi: 10.1038/s41591-018-0106-7. Epub 2018 Jul 16. Nat Med. 2018. PMID: 30013199 Free PMC article.
Stable human FIX expression after 0.9G intrauterine gene transfer of self-complementary adeno-associated viral vector 5 and 8 in macaques.
Mattar CN, Nathwani AC, Waddington SN, Dighe N, Kaeppel C, Nowrouzi A, Mcintosh J, Johana NB, Ogden B, Fisk NM, Davidoff AM, David A, Peebles D, Valentine MB, Appelt JU, von Kalle C, Schmidt M, Biswas A, Choolani M, Chan JK. Mattar CN, et al. Mol Ther. 2011 Nov;19(11):1950-60. doi: 10.1038/mt.2011.107. Epub 2011 May 31. Mol Ther. 2011. PMID: 21629224 Free PMC article.
Pre- and postnatal transplantation of fetal mesenchymal stem cells in osteogenesis imperfecta: a two-center experience.
Götherström C, Westgren M, Shaw SW, Aström E, Biswas A, Byers PH, Mattar CN, Graham GE, Taslimi J, Ewald U, Fisk NM, Yeoh AE, Lin JL, Cheng PJ, Choolani M, Le Blanc K, Chan JK. Götherström C, et al. Stem Cells Transl Med. 2014 Feb;3(2):255-64. doi: 10.5966/sctm.2013-0090. Epub 2013 Dec 16. Stem Cells Transl Med. 2014. PMID: 24342908 Free PMC article. Clinical Trial.
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. Dighe NM, et al. Among authors: mattar cnz. Exp Hematol. 2018 Jun;62:45-55. doi: 10.1016/j.exphem.2018.03.006. Epub 2018 Mar 29. Exp Hematol. 2018. PMID: 29605545 Free PMC article.
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: mattar cnz. 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.
49 results