Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10.

Autor: Maeder ML; Editas Medicine, Cambridge, MA, USA. morganLmaeder@gmail.com., Stefanidakis M; Editas Medicine, Cambridge, MA, USA., Wilson CJ; Editas Medicine, Cambridge, MA, USA., Baral R; Editas Medicine, Cambridge, MA, USA., Barrera LA; Editas Medicine, Cambridge, MA, USA., Bounoutas GS; Editas Medicine, Cambridge, MA, USA., Bumcrot D; Editas Medicine, Cambridge, MA, USA., Chao H; Editas Medicine, Cambridge, MA, USA., Ciulla DM; Editas Medicine, Cambridge, MA, USA., DaSilva JA; Editas Medicine, Cambridge, MA, USA., Dass A; Editas Medicine, Cambridge, MA, USA., Dhanapal V; Editas Medicine, Cambridge, MA, USA., Fennell TJ; Fulcrum Genomics, Somerville, MA, USA., Friedland AE; Editas Medicine, Cambridge, MA, USA., Giannoukos G; Editas Medicine, Cambridge, MA, USA., Gloskowski SW; Editas Medicine, Cambridge, MA, USA., Glucksmann A; Editas Medicine, Cambridge, MA, USA., Gotta GM; Editas Medicine, Cambridge, MA, USA., Jayaram H; Editas Medicine, Cambridge, MA, USA., Haskett SJ; Editas Medicine, Cambridge, MA, USA., Hopkins B; Editas Medicine, Cambridge, MA, USA., Horng JE; Editas Medicine, Cambridge, MA, USA., Joshi S; Editas Medicine, Cambridge, MA, USA., Marco E; Editas Medicine, Cambridge, MA, USA., Mepani R; Editas Medicine, Cambridge, MA, USA., Reyon D; Editas Medicine, Cambridge, MA, USA., Ta T; Editas Medicine, Cambridge, MA, USA., Tabbaa DG; Editas Medicine, Cambridge, MA, USA., Samuelsson SJ; Editas Medicine, Cambridge, MA, USA., Shen S; Editas Medicine, Cambridge, MA, USA., Skor MN; Editas Medicine, Cambridge, MA, USA., Stetkiewicz P; Editas Medicine, Cambridge, MA, USA., Wang T; Editas Medicine, Cambridge, MA, USA., Yudkoff C; Editas Medicine, Cambridge, MA, USA., Myer VE; Editas Medicine, Cambridge, MA, USA., Albright CF; Editas Medicine, Cambridge, MA, USA., Jiang H; Editas Medicine, Cambridge, MA, USA.

Publikováno v: Nature medicine [Nat Med] 2019 Feb; Vol. 25 (2), pp. 229-233. Date of Electronic Publication: 2019 Jan 21.

Tento výsledek nelze pro nepřihlášené uživatele zobrazit.
K zobrazení výsledku je třeba se přihlásit.

Tento výsledek nelze pro nepřihlášené uživatele zobrazit.
K zobrazení výsledku je třeba se přihlásit.

ABE8e Corrects Pax6-Aniridic Variant in Humanized Mouse ESCs and via LNPs in Ex Vivo Cortical Neurons.

Autor: Adair, Bethany A.^1,2 (AUTHOR), Korecki, Andrea J.¹ (AUTHOR), Djaksigulova, Diana¹ (AUTHOR), Wagner, Pamela K.³ (AUTHOR), Chiu, Nina Y.^1,2 (AUTHOR), Lam, Siu Ling¹ (AUTHOR), Lengyell, Tess C.¹ (AUTHOR), Leavitt, Blair R.^1,2,3 (AUTHOR), Simpson, Elizabeth M.^1,2 (AUTHOR) simpson@cmmt.ubc.ca

Publikováno v: Ophthalmology & Therapy. Aug2023, Vol. 12 Issue 4, p2049-2068. 20p.

Impaired adaptation to repeated restraint and decreased response to cold in urocortin 1 knockout mice.

Autor: Zalutskaya AA; Endocrine Unit, Massachusetts General Hospital, Thier 1051, 55 Fruit St., Boston, MA 02114, USA. azalutskaya@partners.org, Arai M, Bounoutas GS, Abou-Samra AB

Publikováno v: American journal of physiology. Endocrinology and metabolism [Am J Physiol Endocrinol Metab] 2007 Jul; Vol. 293 (1), pp. E259-63. Date of Electronic Publication: 2007 Apr 24.