AclandG.M., AguirreG.D., RayJ., et al. (2001). Gene therapy restores vision in a canine model of childhood blindness. Nat. Genet., 28, 92–95.
2.
AclandG.M., AguirreG.D., BennettJ., et al. (2005). Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness. Mol. Ther., 12, 1072–1082.
3.
AdamusG., SugdenB., ShiragaS., et al. (2003). Anti-apoptotic effects of CNTF gene transfer on photoreceptor degeneration in experimental antibody-induced retinopathy. J. Autoimmun., 21, 121–129.
4.
AguirreG.K., KomaromyA.M., CideciyanA.V., et al. (2007). Canine and human visual cortex intact and responsive despite early retinal blindness from RPE65 mutation. PLoS Med., 4, e230.
5.
AlexanderJ.J., UminoY., EverhartD., et al. (2007). Restoration of cone vision in a mouse model of achromatopsia. Nat. Med., 13, 685–687.
6.
AliR.R., ReichelM.B., ThrasherA.J., et al. (1996). Gene transfer into the mouse retina mediated by an adeno-associated viral vector. Hum. Mol. Genet., 5, 591–594.
7.
BaehrW., KaranS., MaedaT., et al. (2007). The function of guanylate cyclase 1 and guanylate cyclase 2 in rod and cone photoreceptors. J. Biol. Chem., 282, 8837–8847.
8.
BaninE., Bandah-RozenfeldD., ObolenskyA., et al. (2010). Molecular anthropology meets genetic medicine to treat blindness in the North African Jewish population: human gene therapy initiated in Israel. Hum. Gene Ther., 21, 1749–1757.
9.
BattenM.L., ImanishiY., TuD.C., et al. (2005). Pharmacological and rAAV gene therapy rescue of visual functions in a blind mouse model of Leber congenital amaurosis. PLoS Med., 2, e333.
10.
BeltranW.A., CideciyanA.V., LewinA.S., et al. (2012). Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa. Proc. Natl. Acad. Sci. USA, 109, 2132–2137.
11.
BennettJ., DuanD., EngelhardtJ.F., and MaguireA.M. (1997). Real-time, noninvasive in vivo assessment of adeno-associated virus-mediated retinal transduction. Invest. Ophthalmol. Vis. Sci., 38, 2857–2863.
12.
BernsK.I. (2013). My life with adeno-associated virus: a long time spent studying a short genome. DNA Cell Biol., 32, 342–347.
13.
BernsK.I., HauswirthW.W., FifeK.H., and LusbyE. (1979). Adeno-associated virus DNA replication. Cold Spring Harb. Symp. Quant. Biol., 43Pt 2, 781–787.
14.
BoyeS.E., BoyeS.L., PangJ., et al. (2010). Functional and behavioral restoration of vision by gene therapy in the guanylate cyclase-1 (GC1) knockout mouse. PLoS One, 5, e11306.
15.
BoyeS.L., ConlonT., ErgerK., et al. (2011). Long-term preservation of cone photoreceptors and restoration of cone function by gene therapy in the guanylate cyclase-1 knockout (GC1KO) mouse. Invest. Ophthalmol. Vis. Sci., 52, 7098–7108.
16.
BoyeS.E., BoyeS.L., LewinA.S., and HauswirthW.W. (2013a). A comprehensive review of retinal gene therapy. Mol. Ther., 21, 509–519.
17.
BoyeS.L., PeshenkoI.V., HuangW.C., et al. (2013b). AAV-mediated gene therapy in the guanylate cyclase (RetGC1/RetGC2) double knockout mouse model of Leber congenital amaurosis. Hum. Gene Ther., 24, 189–202.
18.
BramallA.N., SzegoM.J., PacioneL.R., et al. (2013). Endothelin-2-mediated protection of mutant photoreceptors in inherited photoreceptor degeneration. PLoS One, 8, e58023.
19.
ChangD.D., HauswirthW.W., and ClaytonD.A. (1985). Replication priming and transcription initiate from precisely the same site in mouse mitochondrial DNA. EMBO J., 4, 1559–1567.
20.
ChengL., SapiehaP., KittlerovaP., et al. (2002). TrkB gene transfer protects retinal ganglion cells from axotomy-induced death in vivo. J. Neurosci., 22, 3977–3986.
21.
CheungA.K., HogganM.D., HauswirthW.W., and BernsK.I. (1980). Integration of the adeno-associated virus genome into cellular DNA in latently infected human Detroit 6 cells. J. Virol., 33, 739–748.
22.
CideciyanA.V., AlemanT.S., BoyeS.L., et al. (2008). Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics. Proc. Natl. Acad. Sci. USA, 105, 15112–15117.
23.
CideciyanA.V., HauswirthW.W., AlemanT.S., et al. (2009a). Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year. Hum. Gene Ther., 20, 999–1004.
24.
CideciyanA.V., HauswirthW.W., AlemanT.S., et al. (2009b). Vision 1 year after gene therapy for Leber's congenital amaurosis. N. Engl. J. Med., 361, 725–727.
25.
CideciyanA.V., JacobsonS.G., BeltranW.A., et al. (2013). Human gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement. Proc. Natl. Acad. Sci. USA, 110, 517–525.
26.
ConlonT.J., DengW.T., ErgerK., et al. (2013). Preclinical potency and safety studies of an AAV2-mediated gene therapy vector for the treatment of MERTK associated retinitis pigmentosa. Hum. Gene Ther. Clin. Dev., 24, 23–28.
DalkaraD., ByrneL.C., KlimczakR.R., et al. (2013). In vivo-directed evolution of a new adeno-associated virus for therapeutic outer retinal gene delivery from the vitreous. Sci. Transl. Med., 5, 189ra76.
29.
DanielsM., and HauswirthW. (1971). Fluorescence of the purine and pyrimidine bases of the nucleic acids in neutral aqueous solution at 300 degrees K. Science, 171, 675–677.
30.
DayaS., and BernsK.I. (2008). Gene therapy using adeno-associated virus vectors. Clin. Microbiol. Rev., 21, 583–593.
31.
den HollanderA.I., RoepmanR., KoenekoopR.K., and CremersF.P. (2008). Leber congenital amaurosis: genes, proteins and disease mechanisms. Prog. Retin. Eye Res., 27, 391–419.
32.
DengW.T., SakuraiK., LiuJ., et al. (2009). Functional interchangeability of rod and cone transducin alpha-subunits. Proc. Natl. Acad. Sci. USA, 106, 17681–17686.
33.
DengW.T., DinculescuA., LiQ., et al. (2012). Tyrosine-mutant AAV8 delivery of human MERTK provides long-term retinal preservation in RCS rats. Invest. Ophthalmol. Vis. Sci., 53, 1895–1904.
34.
DengW.T., SakuraiK., KolandaiveluS., et al. (2013). Cone phosphodiesterase-6alpha′ restores rod function and confers distinct physiological properties in the rod phosphodiesterase-6beta-deficient rd10 mouse. J. Neurosci., 33, 11745–11753.
35.
DinculescuA., EstreicherJ., ZentenoJ.C., et al. (2012). Gene therapy for retinitis pigmentosa caused by MFRP mutations: human phenotype and preliminary proof of concept. Hum. Gene Ther., 23, 367–376.
36.
DoroudchiM.M., GreenbergK.P., LiuJ., et al. (2011). Virally delivered channelrhodopsin-2 safely and effectively restores visual function in multiple mouse models of blindness. Mol. Ther., 19, 1220–1229.
37.
DrenserK.A., TimmersA.M., HauswirthW.W., and LewinA.S. (1998). Ribozyme-targeted destruction of RNA associated with autosomal-dominant retinitis pigmentosa. Invest. Ophthalmol. Vis. Sci., 39, 681–689.
38.
DridiS., HiranoY., TaralloV., et al. (2012). ERK1/2 activation is a therapeutic target in age-related macular degeneration. Proc. Natl. Acad. Sci. USA, 109, 13781–13786.
39.
DykaF.M., BoyeS.L., ChiodoV., et al. (2014). Dual AAV vectors result in efficient in vitro and in vivo expression of an oversized gene, MYO7A. Hum. Gene Ther. Methods., 25, 166–177.
40.
FlanneryJ.G., ZolotukhinS., VaqueroM.I., et al. (1997). Efficient photoreceptor-targeted gene expression in vivo by recombinant adeno-associated virus. Proc. Natl. Acad. Sci. USA, 94, 6916–6921.
41.
GeneadM.A., FishmanG.A., RhaJ., et al. (2011). Photoreceptor structure and function in patients with congenital achromatopsia. Invest. Ophthalmol. Vis. Sci., 52, 7298–7308.
42.
GhivizzaniS.C., MackayS.L., MadsenC.S., et al. (1993). Transcribed heteroplasmic repeated sequences in the porcine mitochondrial DNA D-loop region. J. Mol. Evol., 37, 36–37.
43.
GorbatyukM.S., KnoxT., LaVailM.M., et al. (2010). Restoration of visual function in P23H rhodopsin transgenic rats by gene delivery of BiP/Grp78. Proc. Natl. Acad. Sci. USA, 107, 5961–5966.
44.
GuyJ., QiX., and HauswirthW.W. (1998). Adeno-associated viral-mediated catalase expression suppresses optic neuritis in experimental allergic encephalomyelitis. Proc. Natl. Acad. Sci. USA, 95, 13847–13852.
45.
GuyJ., QiX., KoilkondaR.D., et al. (2009). Efficiency and safety of AAV-mediated gene delivery of the human ND4 complex I subunit in the mouse visual system. Invest. Ophthalmol. Vis. Sci., 50, 4205–4214.
46.
GuziewiczK.E., ZangerlB., KomaromyA.M., et al. (2013). Recombinant AAV-mediated BEST1 transfer to the retinal pigment epithelium: analysis of serotype-dependent retinal effects. PLoS One, 8, e75666.
47.
HauswirthW.W., and BernsK.I. (1977). Origin and termination of adeno-associated virus DNA replication. Virology, 78, 488–499.
48.
HauswirthW.W., and BernsK.I. (1979). Adeno-associated virus DNA replication: nonunit-length molecules. Virology, 93, 57–68.
49.
HauswirthW.W., and ClaytonD.A. (1985). Length heterogeneity of a conserved displacement-loop sequence in human mitochondrial DNA. Nucleic Acids Res., 13, 8093–8104.
50.
HauswirthW.W., and LaipisP.J. (1982). Mitochondrial DNA polymorphism in a maternal lineage of Holstein cows. Proc. Natl. Acad. Sci. USA, 79, 4686–4690.
51.
HauswirthW., and WangS.Y. (1973). Pyrimidine adduct fluorescence in UV irradiated nucleic acids. Biochem. Biophys. Res. Commun., 51, 819–826.
52.
HauswirthW., and WangS.Y. (1977a). Cytidine-C(5)-photoexchange: a kinetic analysis. Photochem. Photobiol., 26, 231–234.
53.
HauswirthW., and WangS.Y. (1977b). Excited state processes and solution conformation of dipyrimidine adducts. Photochem. Photobiol., 25, 161–166.
54.
HauswirthW., HahnB.S., and WangS.Y. (1972). Spontaneous and light induced hydration of pyrimidines. Biochem. Biophys. Res. Commun., 48, 1614–1621.
55.
HauswirthW.W., LaipisP.J., GilmanM.E., et al. (1980). Genetic mapping of bovine mitochondrial DNA from a single animal. Gene, 8, 193–209.
56.
HauswirthW.W., Van de WalleM.J., LaipisP.J., and OlivoP.D. (1984). Heterogeneous mitochondrial DNA D-loop sequences in bovine tissue. Cell, 37, 1001–1007.
57.
HauswirthW.W., AlemanT.S., KaushalS., et al. (2008). Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial. Hum. Gene Ther., 19, 979–990.
58.
HumphriesP., KennaP., and FarrarG.J. (1992). On the molecular genetics of retinitis pigmentosa. Science, 256, 804–808.
59.
JacobsonS.G., CideciyanA.V., PeshenkoI.V., et al. (2012). Determining the consequences of retinal membrane guanylyl cyclase (RetGC) deficiency in human Leber congenital amaurosis en route to therapy: residueal cone photoreceptor vision correlates with biocheminal properties of the mutants. Hum. Mol. Genet., 22, 168–183.
60.
JanssenA., MinS.H., MoldayL.L., et al. (2008). Effect of late-stage therapy on disease progression in AAV-mediated rescue of photoreceptor cells in the retinoschisin-deficient mouse. Mol. Ther., 16, 1010–1017.
61.
JiangL., LiT.Z., BoyeS.E., et al. (2013). RNAi-mediated gene suppression in a GCAP1(L151F) cone-rod dystrophy mouse model. PLoS One, 8, e57676.
62.
KhattakM.N., HauswirthW., and WangS.Y. (1972). Photohydration of pyrimidines in “acid puddles.”. Biochem. Biophys. Res. Commun., 48, 1622–1629.
63.
KoilkondaR.D., YuH., ChouT.H., et al. (2014). Safety and effects of the vector for the Leber hereditary optic neuropathy gene therapy clinical trial. JAMA Ophthalmol., 132, 409–420.
64.
KomaromyA.M., AlexanderJ.J., RowlanJ.S., et al. (2010). Gene therapy rescues cone function in congenital achromatopsia. Hum. Mol. Genet., 19, 2581–2593.
65.
KuC.A., ChiodoV.A., BoyeS.L., et al. (2011). Gene therapy using self-complementary Y733F capsid mutant AAV2/8 restores vision in a model of early onset Leber congenital amaurosis. Hum. Mol. Genet., 20, 4569–4581.
66.
KwonB.K., LiuJ., LamC., et al. (2007). Brain-derived neurotrophic factor gene transfer with adeno-associated viral and lentiviral vectors prevents rubrospinal neuronal atrophy and stimulates regeneration-associated gene expression after acute cervical spinal cord injury. Spine (Phila Pa 1976), 32, 1164–1173.
67.
LaipisP.J., HauswirthW.W., O'BrienT.W., and MichaelsG.S. (1979). A physical map of bovine mitochondrial DNA from a single animal. Biochim. Biophys. Acta, 565, 22–32.
68.
LeonardK.C., PetrinD., CouplandS.G., et al. (2007). XIAP protection of photoreceptors in animal models of retinitis pigmentosa. PLoS One, 2, e314.
69.
LewinA.S., DrenserK.A., HauswirthW.W., et al. (1998). Ribozyme rescue of photoreceptor cells in a transgenic rat model of autosomal dominant retinitis pigmentosa. Nat. Med., 4, 967–971.
70.
LiQ., DinculescuA., ShanZ., et al. (2008). Downregulation of p22phox in retinal pigment epithelial cells inhibits choroidal neovascularization in mice. Mol. Ther., 16, 1688–1694.
71.
LiX., LiW., DaiX., et al. (2011). Gene therapy rescues cone structure and function in the 3-month-old rd12 mouse: a model for midcourse RPE65 leber congenital amaurosis. Invest. Ophthalmol. Vis. Sci., 52, 7–15.
72.
LopesV.S., BoyeS.E., LouieC.M., et al. (2013). Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus. Gene Ther., 20, 824–833.
73.
LukasonM., DuFresneE., RubinH., et al. (2011). Inhibition of choroidal neovascularization in a nonhuman primate model by intravitreal administration of an AAV2 vector expressing a novel anti-VEGF molecule. Mol. Ther., 19, 260–265.
74.
MadsenC.S., GhivizzaniS.C., and HauswirthW.W. (1993). In vivo and in vitro evidence for slipped mispairing in mammalian mitochondria. Proc. Natl. Acad. Sci. USA, 90, 7671–7675.
75.
MancusoK., NeitzM., and NeitzJ. (2006). An adaptation of the Cambridge Colour Test for use with animals. Vis. Neurosci., 23, 695–701.
76.
MancusoK., HendricksonA.E., ConnorT.B.Jr., et al. (2007). Recombinant adeno-associated virus targets passenger gene expression to cones in primate retina. J. Opt. Soc. Am. A, 24, 1411–1416.
77.
MancusoK., HauswirthW.W., LiQ., et al. (2009). Gene therapy for red-green colour blindness in adult primates. Nature, 461, 784–787.
78.
MaoH., JamesT.Jr., SchweinA., et al. (2011). AAV delivery of wild-type rhodopsin preserves retinal function in a mouse model of autosomal dominant retinitis pigmentosa. Hum. Gene Ther., 22, 567–575.
79.
MartinK.R., QuigleyH.A., ZackD.J., et al. (2003). Gene therapy with brain-derived neurotrophic factor as a protection: retinal ganglion cells in a rat glaucoma model. Invest. Ophthalmol. Vis. Sci., 44, 4357–4365.
80.
McGee SanftnerL.H., AbelH., HauswirthW.W., and FlanneryJ.G. (2001). Glial cell line derived neurotrophic factor delays photoreceptor degeneration in a transgenic rat model of retinitis pigmentosa. Mol. Ther., 4, 622–629.
81.
MinS.H., MoldayL.L., SeeligerM.W., et al. (2005). Prolonged recovery of retinal structure/function after gene therapy in an Rs1h-deficient mouse model of x-linked juvenile retinoschisis. Mol. Ther., 12, 644–651.
82.
MoldayL.L., DjajadiH., YanP., et al. (2013). RD3 gene delivery restores guanylate cyclase localization and rescues photoreceptors in the Rd3 mouse model of Leber congenital amaurosis 12. Hum. Mol. Genet., 22, 3894–3905.
83.
MollonJ.D., BowmakerJ.K., and JacobsG.H. (1984). Variations of colour vision in a New World primate can be explained by polymorphism of retinal photopigments. Proc. R. Soc. Lond. B, 222, 373–399.
84.
MoriK., GehlbachP., YamamotoS., et al. (2002). AAV-mediated gene transfer of pigment epithelium-derived factor inhibits choroidal neovascularization. Invest. Ophthalmol. Vis. Sci., 43, 1994–2000.
85.
OlivoP.D., Van de WalleM.J., LaipisP.J., and HauswirthW.W. (1983). Nucleotide sequence evidence for rapid genotypic shifts in the bovine mitochondrial DNA D-loop. Nature, 306, 400–402.
86.
PangJ.J., ChangB., KumarA., et al. (2006). Gene therapy restores vision-dependent behavior as well as retinal structure and function in a mouse model of RPE65 Leber congenital amaurosis. Mol. Ther., 13, 565–572.
87.
PangJ.J., BoyeS.L., KumarA., et al. (2008). AAV-mediated gene therapy for retinal degeneration in the rd10 mouse containing a recessive PDEbeta mutation. Invest. Ophthalmol. Vis. Sci., 49, 4278–4283.
88.
PangJ.J., DengW.T., DaiX., et al. (2012). AAV-mediated cone rescue in a naturally occurring mouse model of CNGA3-achromatopsia. PLoS One, 7, e35250.
89.
PasadhikaS., FishmanG.A., StoneE.M., et al. (2010). Differential macular morphology in patients with RPE65-, CEP290-, GUCY2D-, and AIPL1-related Leber congenital amaurosis. Invest. Ophthalmol. Vis. Sci., 51, 2608–2614.
90.
PechanP., RubinH., LukasonM., et al. (2009). Novel anti-VEGF chimeric molecules delivered by AAV vectors for inhibition of retinal neovascularization. Gene Ther., 16, 10–16.
91.
PetrinD., BakerA., BrousseauJ., et al. (2003). XIAP protects photoreceptors from n-methyl-n-nitrosourea-induced retinal degeneration. Adv. Exp. Med. Biol., 533, 385–393.
92.
Petrs-SilvaH., DinculescuA., LiQ., et al. (2009). High-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors. Mol. Ther., 17, 463–471.
93.
Petrs-SilvaH., DinculescuA., LiQ., et al. (2011). Novel properties of tyrosine-mutant AAV2 vectors in the mouse retina. Mol. Ther., 19, 293–301.
94.
QiX., LewinA.S., SunL., et al. (2004). SOD2 gene transfer protects against optic neuropathy induced by deficiency of complex I. Ann. Neurol., 56, 182–191.
95.
QiX., LewinA.S., SunL., et al. (2007a). Suppression of mitochondrial oxidative stress provides long-term neuroprotection in experimental optic neuritis. Invest. Ophthalmol. Vis. Sci., 48, 681–691.
96.
QiX., SunL., HauswirthW.W., et al. (2007b). Use of mitochondrial antioxidant defenses for rescue of cells with a Leber hereditary optic neuropathy-causing mutation. Arch. Ophthalmol., 125, 268–272.
97.
QiX., SunL., LewinA.S., et al. (2007c). Long-term suppression of neurodegeneration in chronic experimental optic neuritis: antioxidant gene therapy. Invest. Ophthalmol. Vis. Sci., 48, 5360–5370.
98.
QiX., SunL., LewinA.S., et al. (2007d). The mutant human ND4 subunit of complex I induces optic neuropathy in the mouse. Invest. Ophthalmol. Vis. Sci., 48, 1–10.
99.
RaislerB.J., BernsK.I., GrantM.B., et al. (2002). Adeno-associated virus type-2 expression of pigmented epithelium-derived factor or Kringles 1–3 of angiostatin reduce retinal neovascularization. Proc. Natl. Acad. Sci. USA, 99, 8909–8914.
100.
RamamurthyV., NiemiG.A., RehT.A., and HurleyJ.B. (2004). Leber congenital amaurosis linked to AIPL1: a mouse model reveals destabilization of cGMP phosphodiesterase. Proc. Natl. Acad. Sci. USA, 101, 13897–13902.
101.
RenwickJ., NarangM.A., CouplandS.G., et al. (2006). XIAP-mediated neuroprotection in retinal ischemia. Gene Ther., 13, 339–347.
102.
RheeK.D., RuizA., DuncanJ.L., et al. (2007). Molecular and cellular alterations induced by sustained expression of ciliary neurotrophic factor in a mouse model of retinitis pigmentosa. Invest. Ophthalmol. Vis. Sci., 48, 1389–1400.
103.
RomanA.J., BoyeS.L., AlemanT.S., et al. (2007). Electroretinographic analyses of Rpe65-mutant rd12 mice: developing an in vivo bioassay for human gene therapy trials of Leber congenital amaurosis. Mol. Vis., 13, 1701–1710.
104.
RosenfeldP.J., CowleyG.S., McGeeT.L., et al. (1992). A null mutation in the rhodopsin gene causes rod photoreceptor dysfunction and autosomal recessive retinitis pigmentosa. Nat. Genet., 1, 209–213.
105.
SamulskiR.J., BernsK.I., TanM., and MuzyczkaN. (1982). Cloning of adeno-associated virus into pBR322: rescue of intact virus from the recombinant plasmid in human cells. Proc. Natl. Acad. Sci. USA, 79, 2077–2081.
106.
SamulskiR.J., SrivastavaA., BernsK.I., and MuzyczkaN. (1983). Rescue of adeno-associated virus from recombinant plasmids: gene correction within the terminal repeats of AAV. Cell, 33, 135–143.
107.
SapiehaP.S., PeltierM., RendahlK.G., et al. (2003). Fibroblast growth factor-2 gene delivery stimulates axon growth by adult retinal ganglion cells after acute optic nerve injury. Mol. Cell Neurosci., 24, 656–672.
108.
SimonsD.L., BoyeS.L., HauswirthW.W., and WuS.M. (2011). Gene therapy prevents photoreceptor death and preserves retinal function in a Bardet-Biedl syndrome mouse model. Proc. Natl. Acad. Sci. USA, 108, 6276–6281.
109.
SpearI.S., FifeK.H., HauswirthW.W., et al. (1977). Evidence for two nucleotide sequence orientations within the terminal repetition of adeno-associated virus DNA. J. Virol., 24, 627–634.
110.
TimmersA.M., NewtonB.R., and HauswirthW.W. (1993). Synthesis and stability of retinal photoreceptor mRNAs are coordinately regulated during bovine fetal development. Exp. Eye Res., 56, 257–265.
TrapaniI., ColellaP., SommellaA., et al. (2014). Effective delivery of large genes to the retina by dual AAV vectors. EMBO Mol. Med., 6, 194–211.
113.
van GinkelP.R., and HauswirthW.W. (1994). Parallel regulation of fetal gene expression in different photoreceptor cell types. J. Biol. Chem., 269, 4986–4992.
114.
van GinkelP.R., TimmersA.M., SzelA., and HauswirthW.W. (1995). Topographical regulation of cone and rod opsin genes: parallel, position dependent levels of transcription. Brain Res. Dev. Brain Res., 89, 146–149.
115.
YaoJ., FeathersK.L., KhannaH., et al. (2011). XIAP therapy increases survival of transplanted rod precursors in a degenerating host retina. Invest. Ophthalmol. Vis. Sci., 52, 1567–1572.
116.
YuH., OzdemirS.S., KoilkondaR.D., et al. (2012). Mutant NADH dehydrogenase subunit 4 gene delivery to mitochondria by targeting sequence-modified adeno-associated virus induces visual loss and optic atrophy in mice. Mol. Vis., 18, 1668–1683.
117.
YuH., MehtaA., WangG., et al. (2013). Next-generation sequencing of mitochondrial targeted AAV transfer of human ND4 in mice. Mol. Vis., 19, 1482–1491.
118.
Zadro-LamoureuxL.A., ZacksD.N., BakerA.N., et al. (2009). XIAP effects on retinal detachment-induced photoreceptor apoptosis [corrected]. Invest. Ophthalmol. Vis. Sci., 50, 1448–1453.
119.
ZhongL., LiB., JayandharanG., et al. (2008a). Tyrosine-phosphorylation of AAV2 vectors and its consequences on viral intracellular trafficking and transgene expression. Virology, 381, 194–202.
120.
ZhongL., LiB., MahC.S., et al. (2008b). Next generation of adeno-associated virus 2 vectors: point mutations in tyrosines lead to high-efficiency transduction at lower doses. Proc. Natl. Acad. Sci. USA, 105, 7827–7832.
121.
ZouJ., LuoL., ShenZ., et al. (2011). Whirlin replacement restores the formation of the USH2 protein complex in whirlin knockout photoreceptors. Invest. Ophthalmol. Vis. Sci., 52, 2343–2351.
