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Terapia Génica en Distrofias Retinales Hereditarias
1.
Yanping Yang and Christopher E. Walsh, Spliceosome-
Mediated RNA Trans-Splicing, Molecular Therapy
(2005) 12, 1006–1012
2.
Vecino E, Terapia génica contra la retinosis pigmentaria,
ArchSocEspOftalmol 2008; 83: 213-214
3.
Study of UshStat in Patients With Retinitis Pigmentosa
Associated With Usher Syndrome Type 1B Principal
Investigator: Richard Weleber, MD Casey Eye Institute,
Portland, OregonJose-Alain Sahel, MD, PhD Hopital
Nationale des Quinze-Vingt, Paris France.
4.
Daniel C. Chung, Vivian Lee and Albert M. Maguire,
Recent advances in ocular gene therapy. Current
Opinion in Ophthalmology 2009,20:377–381
5.
Safety and Efficacy Study in Subjects With Leber
Congenital Amaurosis. Clinical Trials Fase III. 24
pacientes. University Iowa Philadelphia Principal
Investigator: Albert MMaguire, MDChildren’s Hospital
of Philadelphia Stephen R Russell, MDUniversity of Iowa
6.
J Kong et al, Correction of the disease phenotype in the
mousemodel of Stargardt disease by lentiviral gene therapy.
Gene Therapy (2008) 15, 1311–1320
7.
Phase I/IIa Study of StarGen in Patients With Stargardt
Macular Degeneration David Wilson, MDOregon
Health & Science University, Portland, Oregon Jose-
Alain Sahel, MD. Ph.D Hopital Nationale des Quinze-
Vingt, Paris France
8.
Shannon E Boye et al, A Comprehensive Review of Retinal
Gene Therapy, Mol There 2013 Mar, 21(3) 509-519
9.
Mac Laren, R et al Retinal gene therapy in patients
with choroideremia: Initial findings from a phase 1/2
clinical trial”. The Lancet 383 (9923)
BIBLIOGRAFÍA