Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa

^aSection of Ophthalmology, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104;
^bDepartment of Ophthalmology, Scheie Eye Institute, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA 19104;
^cDepartment of Molecular Genetics and Microbiology, University of Florida, Gainesville, FL 32610;
^dDepartment of Ophthalmology and Visual Sciences, University of Michigan, Ann Arbor, MI 48105;
^eDepartment of Ophthalmology, University of Massachusetts Medical School, Worcester, MA 01605;
^fDepartment of Ophthalmology, University of Florida, Gainesville, FL 32610; and
^gNeurobiology-Neurodegeneration and Repair Laboratory, National Eye Institute, National Institutes of Health, Bethesda, MD 20892

Edited by Jeremy Nathans, The Johns Hopkins University, Baltimore, MD, and approved December 20, 2011 (received for review November 16, 2011)

Footnotes

↵¹To whom correspondence may be addressed. E-mail: wbeltran{at}vet.upenn.edu or gda{at}vet.upenn.edu.

Author contributions: W.A.B., A.V.C., S.G.J., and G.D.A. designed research; W.A.B., A.V.C., A.S.L., S.I., A. Sumaroka, A.J.R., M.S., T.S.A., S.G., A. Swaroop, W.W.H., S.G.J., and G.D.A. performed research; A.S.L., H.K., A. Sumaroka, V.A.C., D.S.F., W.-T.D., S.L.B., A. Swaroop, and W.W.H. contributed new reagents/analytic tools; W.A.B., A.V.C., A. Sumaroka, A.J.R., M.S., S.G.J., and G.D.A. analyzed data; and W.A.B., A.V.C., S.G.J., and G.D.A. wrote the paper.
Conflict of interest statement: The authors declare a conflict of interest. W.W.H. and the University of Florida have a financial interest in the use of adeno-associated virus therapies and own equity in a company (AGTC Inc.) that might, in the future, commercialize some aspects of this work. The remaining authors declare no conflict of interest.
This article is a PNAS Direct Submission.
This article contains supporting information online at www.pnas.org/lookup/suppl/doi:10.1073/pnas.1118847109/-/DCSupplemental.

Cited By...

Article usage

Article usage: January 2012 to April 2021

	Abstract	Full	Pdf
Jan 2012	2472	0	276
Feb 2012	3786	241	565
Mar 2012	1017	129	202
Apr 2012	614	125	148
May 2012	504	92	98
Jun 2012	328	85	93
Jul 2012	263	39	63
Aug 2012	212	45	74
Sep 2012	272	64	78
Oct 2012	238	83	103
Nov 2012	233	81	82
Dec 2012	229	59	64
Total 2012	10168	1043	1846
Jan 2013	265	72	94
Feb 2013	264	65	117
Mar 2013	246	61	86
Apr 2013	320	78	88
May 2013	163	40	33
Jun 2013	152	46	30
Jul 2013	172	38	35
Aug 2013	106	28	168
Sep 2013	150	38	58
Oct 2013	167	36	56
Nov 2013	191	42	52
Dec 2013	165	30	43
Total 2013	2361	574	860
Jan 2014	169	81	45
Feb 2014	136	58	35
Mar 2014	216	65	38
Apr 2014	157	49	41
May 2014	143	36	34
Jun 2014	99	25	19
Jul 2014	140	29	20
Aug 2014	143	54	31
Sep 2014	206	69	29
Oct 2014	199	78	49
Nov 2014	199	49	37
Dec 2014	201	46	36
Total 2014	2008	639	414
Jan 2015	205	68	45
Feb 2015	194	86	31
Mar 2015	213	132	42
Apr 2015	133	69	31
May 2015	104	40	18
Jun 2015	104	30	23
Jul 2015	97	15	22
Aug 2015	118	26	14
Sep 2015	115	59	26
Oct 2015	161	95	37
Nov 2015	151	65	48
Dec 2015	121	51	23
Total 2015	1716	736	360
Jan 2016	104	46	19
Feb 2016	111	80	20
Mar 2016	129	54	36
Apr 2016	137	55	42
May 2016	121	52	19
Jun 2016	96	29	17
Jul 2016	99	24	22
Aug 2016	99	32	9
Sep 2016	115	27	12
Oct 2016	121	51	28
Nov 2016	189	71	39
Dec 2016	84	24	18
Total 2016	1405	545	281
Jan 2017	108	42	22
Feb 2017	84	52	18
Mar 2017	156	42	19
Apr 2017	123	39	20
May 2017	159	37	20
Jun 2017	142	71	18
Jul 2017	109	26	10
Aug 2017	125	63	18
Sep 2017	163	42	25
Oct 2017	171	43	22
Nov 2017	151	59	41
Dec 2017	144	158	36
Total 2017	1635	674	269
Jan 2018	157	42	18
Feb 2018	200	130	16
Mar 2018	197	111	28
Apr 2018	153	87	47
May 2018	185	121	15
Jun 2018	116	96	14
Jul 2018	138	96	14
Aug 2018	162	98	15
Sep 2018	140	87	13
Oct 2018	59	59	13
Nov 2018	67	99	43
Dec 2018	52	268	33
Total 2018	1626	1294	269
Jan 2019	43	435	31
Feb 2019	39	182	30
Mar 2019	22	69	19
Apr 2019	26	81	27
May 2019	31	151	27
Jun 2019	20	73	7
Jul 2019	36	41	28
Aug 2019	36	66	40
Sep 2019	41	59	31
Oct 2019	59	84	19
Nov 2019	50	95	34
Dec 2019	58	140	38
Total 2019	461	1476	331
Jan 2020	63	106	44
Feb 2020	45	91	31
Mar 2020	46	73	29
Apr 2020	44	58	21
May 2020	66	133	28
Jun 2020	29	59	19
Jul 2020	14	50	8
Aug 2020	12	69	31
Sep 2020	18	94	25
Oct 2020	26	88	32
Nov 2020	29	91	30
Dec 2020	29	170	47
Total 2020	421	1082	345
Jan 2021	17	94	31
Feb 2021	13	64	26
Mar 2021	28	93	34
Apr 2021	9	51	8
Total 2021	67	302	99
Total	21868	8365	5074