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In vivo topical gene therapy for recessive dystrophic epidermolysis bullosa: a phase 1 and 2 trial.
Gurevich I, Agarwal P, Zhang P, Dolorito JA, Oliver S, Liu H, Reitze N, Sarma N, Bagci IS, Sridhar K, Kakarla V, Yenamandra VK, O'Malley M, Prisco M, Tufa SF, Keene DR, South AP, Krishnan SM, Marinkovich MP. Gurevich I, et al. Among authors: tufa sf. Nat Med. 2022 Apr;28(4):780-788. doi: 10.1038/s41591-022-01737-y. Epub 2022 Mar 28. Nat Med. 2022. PMID: 35347281 Free PMC article. Clinical Trial.
In vivo studies of mutant fibrillin-1 microfibrils.
Charbonneau NL, Carlson EJ, Tufa S, Sengle G, Manalo EC, Carlberg VM, Ramirez F, Keene DR, Sakai LY. Charbonneau NL, et al. J Biol Chem. 2010 Aug 6;285(32):24943-55. doi: 10.1074/jbc.M110.130021. Epub 2010 Jun 7. J Biol Chem. 2010. PMID: 20529844 Free PMC article.
Abnormal Activation of BMP Signaling Causes Myopathy in Fbn2 Null Mice.
Sengle G, Carlberg V, Tufa SF, Charbonneau NL, Smaldone S, Carlson EJ, Ramirez F, Keene DR, Sakai LY. Sengle G, et al. Among authors: tufa sf. PLoS Genet. 2015 Jun 26;11(6):e1005340. doi: 10.1371/journal.pgen.1005340. eCollection 2015 Jun. PLoS Genet. 2015. PMID: 26114882 Free PMC article.
39 results