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Article ; Online: AAV2-Mediated Expression of HspB1 in RGCs Prevents Somal Damage and Axonal Transport Deficits in a Mouse Model of Ocular Hypertension.

Nam, Mi-Hyun / Nahomi, Rooban B / Pantcheva, Mina B / Dhillon, Armaan / Chiodo, Vince A / Smith, W Clay / Nagaraj, Ram H

Translational vision science & technology

2022 Volume 11, Issue 11, Page(s) 8

Abstract: Purpose: Ocular hypertension is a significant risk factor for vision loss in glaucoma caused by the death of retinal ganglion cells (RGCs). We investigated whether small heat shock proteins (sHsps) expressed in RGCs protect those cells against ocular ... ...

Abstract	Purpose: Ocular hypertension is a significant risk factor for vision loss in glaucoma caused by the death of retinal ganglion cells (RGCs). We investigated whether small heat shock proteins (sHsps) expressed in RGCs protect those cells against ocular hypertension in mice. Methods: AAV2 vectors encoding genes for one of the following four human sHsps: HSPB1, HSPB4, HSPB5, or HSPB6 were constructed for RGC-specific expression. Ischemia/reperfusion was induced by elevating the intraocular pressure (IOP) to 120 mm Hg for one hour, followed by a rapid return to normal IOP. Microbeads (MB) were injected into the anterior chamber of mice to induce ocular hypertension. RGC death and glial activation were assessed by immunostaining for Brn3a, RBPMS, Iba1, and glial fibrillary acid protein in retinal flat mounts. RGC axonal defects were evaluated by anterograde transport of intravitreally injected cholera toxin-B. RGC function was assessed by pattern electroretinography. Results: Among the sHsps, HspB1 offered the best protection against RGC death from ischemia/reperfusion injury in the mouse retina. Intravitreal administration of AAV2-HSPB1 either two weeks before or one week after instituting ocular hypertension resulted in significant prevention of RGC loss. The MB-injected mice showed RGC axonal transportation defects, but AAV2-HSPB1 administration significantly inhibited this defect. AAV2-HSPB1 prevented glial activation caused by ocular hypertension. More importantly, a single injection of AAV2-HSPB1 protected RGCs long-term in MB-injected eyes. Conclusions: The administration of AAV2-HSPB1 inhibited RGC death and axonal transport defects and reduced glial activation in a mouse model of ocular hypertension. Translational relevance: Our results suggested that the intravitreal delivery of AAV2-HSPB1 could be developed as a gene therapy to prevent vision loss on a long-term basis in glaucoma patients.
MeSH term(s)	Humans ; Mice ; Animals ; Retinal Ganglion Cells/metabolism ; Axonal Transport ; Ocular Hypertension/genetics ; Ocular Hypertension/metabolism ; Glaucoma/genetics ; Glaucoma/prevention & control ; Intraocular Pressure ; Disease Models, Animal ; Heat-Shock Proteins/genetics ; Heat-Shock Proteins/metabolism ; Molecular Chaperones/genetics ; Molecular Chaperones/metabolism
Chemical Substances	HSPB1 protein, human ; Heat-Shock Proteins ; Molecular Chaperones
Language	English
Publishing date	2022-11-07
Publishing country	United States
Document type	Journal Article ; Research Support, Non-U.S. Gov't
ZDB-ID	2674602-5
ISSN	2164-2591 ; 2164-2591
ISSN (online)	2164-2591
ISSN	2164-2591
DOI	10.1167/tvst.11.11.8
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Article ; Online: Intravitreal Administration of AAV2-SIRT1 Reverses Diabetic Retinopathy in a Mouse Model of Type 2 Diabetes.

Adu-Agyeiwaah, Yvonne / Vieira, Cristiano P / Asare-Bediako, Bright / Li Calzi, Sergio / DuPont, Mariana / Floyd, Jason / Boye, Sanford / Chiodo, Vince / Busik, Julia V / Grant, Maria B

Translational vision science & technology

2023 Volume 12, Issue 4, Page(s) 20

Abstract: Purpose: The expression of silent information regulator (SIRT) 1 is reduced in diabetic retinopathy (DR). Previous studies showed that alterations in SIRT1 messenger RNA (mRNA) and protein expression are implicated in progressive inflammation and ... ...

Abstract	Purpose: The expression of silent information regulator (SIRT) 1 is reduced in diabetic retinopathy (DR). Previous studies showed that alterations in SIRT1 messenger RNA (mRNA) and protein expression are implicated in progressive inflammation and formation of retinal acellular capillaries. Treatment with the SIRT1 agonist, SRT1720, improved visual response by restoration of a- and b-wave responses on electroretinogram scotopic measurements in diabetic (db/db) mice. In this study, we investigated the effects of intravitreal SIRT1 delivery on diabetic retinal pathology. Methods: Nine-month-old db/db mice received one intravitreal injection of either AAV2-SIRT1 or AAV2-GFP control virus, and after 3 months, electroretinography and optomotor responses were measured. Their eyes were then removed and analyzed by immunohistochemistry and flow cytometry. Results: SIRT1 mRNA and protein levels were increased following AAV2-SIRT1 administration compared to control virus AAV2-GFP injected mice. IBA1+ and caspase 3 expression were decreased in retinas of db/db mice injected with AAV2-SIRT1, and reductions in scotopic a- and b-waves and high spatial frequency in optokinetic response were prevented. Retinal hypoxia inducible factor 1α (HIF-1α) protein levels were reduced in the AAV2-SIRT1-injected mice compared to control-injected mice. Using flow cytometry to assess changes in intracellular HIF-1α levels, endothelial cells (CD31+) from AAV-2 SIRT1 injected mice demonstrated reduced HIF-1α expression compared to db/db mice injected with the control virus. Conclusions: Intravitreal AAV2-SIRT1 delivery increased retina SIRT1 and transduced neural and endothelial cells, thus reversing functional damage and improving overall visual function. Translational relevance: AAV2-SIRT1 gene therapy represents a beneficial approach for the treatment of chronic retinal conditions such as DR.
MeSH term(s)	Mice ; Animals ; Diabetic Retinopathy/genetics ; Diabetic Retinopathy/therapy ; Sirtuin 1/genetics ; Diabetes Mellitus, Type 2/complications ; Diabetes Mellitus, Type 2/therapy ; Endothelial Cells/metabolism ; Disease Models, Animal ; RNA, Messenger
Chemical Substances	Sirtuin 1 (EC 3.5.1.-) ; RNA, Messenger ; Sirt1 protein, mouse (EC 3.5.1.-)
Language	English
Publishing date	2023-04-06
Publishing country	United States
Document type	Journal Article ; Research Support, N.I.H., Extramural ; Research Support, Non-U.S. Gov't
ZDB-ID	2674602-5
ISSN	2164-2591 ; 2164-2591
ISSN (online)	2164-2591
ISSN	2164-2591
DOI	10.1167/tvst.12.4.20
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Article: Prdm1 overexpression causes a photoreceptor fate-shift in nascent, but not mature, bipolar cells

Goodson, Noah B / Park, Ko U / Silver, Jason S / Chiodo, Vince A / Hauswirth, William W / Brzezinski, Joseph A

Developmental biology. 2020 Aug. 15, v. 464, no. 2

2020

Abstract: The transcription factors Prdm1 (Blimp1) and Vsx2 (Chx10) work downstream of Otx2 to regulate photoreceptor and bipolar cell fates in the developing retina. Mice that lack Vsx2 fail to form bipolar cells while Prdm1 mutants form excess bipolars at the ... ...

Abstract	The transcription factors Prdm1 (Blimp1) and Vsx2 (Chx10) work downstream of Otx2 to regulate photoreceptor and bipolar cell fates in the developing retina. Mice that lack Vsx2 fail to form bipolar cells while Prdm1 mutants form excess bipolars at the direct expense of photoreceptors. Excess bipolars in Prdm1 mutants appear to derive from rods, suggesting that photoreceptor fate remains mutable for some time after cells become specified. Here we tested whether bipolar cell fate is also plastic during development. To do this, we created a system to conditionally misexpress Prdm1 at different stages of bipolar cell development. We found that Prdm1 blocks bipolar cell formation if expressed before the fate choice decision occurred. When we misexpressed Prdm1 just after the decision to become a bipolar cell was made, some cells were reprogrammed into photoreceptors. In contrast, Prdm1 misexpression in mature bipolar cells did not affect cell fate. We also provide evidence that sustained misexpression of Prdm1 was selectively toxic to photoreceptors. Our data show that bipolar fate is malleable, but only for a short temporal window following fate specification. Prdm1 and Vsx2 act by stabilizing photoreceptor and bipolar fates in developing OTX2+ cells of the retina.
Keywords	mice ; mutants ; photoreceptors ; retina ; toxicity ; transcription factors
Language	English
Dates of publication	2020-0815
Size	p. 111-123.
Publishing place	Elsevier Inc.
Document type	Article
ZDB-ID	1114-9
ISSN	1095-564X ; 0012-1606
ISSN (online)	1095-564X
ISSN	0012-1606
DOI	10.1016/j.ydbio.2020.06.003
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Article ; Online: Dual

Dyka, Frank M / Molday, Laurie L / Chiodo, Vince A / Molday, Robert S / Hauswirth, William W

Human gene therapy

2019 Volume 30, Issue 11, Page(s) 1361–1370

Abstract: Autosomal recessive Stargardt disease is the most common inherited macular degeneration in humans. It is caused by mutations in the retina-specific ATP binding cassette transporter A4 (ABCA4) that is essential for the clearance of all- ...

Abstract	Autosomal recessive Stargardt disease is the most common inherited macular degeneration in humans. It is caused by mutations in the retina-specific ATP binding cassette transporter A4 (ABCA4) that is essential for the clearance of all-
MeSH term(s)	ATP-Binding Cassette Transporters/genetics ; ATP-Binding Cassette Transporters/therapeutic use ; Animals ; Dependovirus/genetics ; Disease Models, Animal ; Fluorescence ; Fundus Oculi ; Genes, Recessive ; Genetic Vectors/metabolism ; HEK293 Cells ; Humans ; Mice, Inbred C57BL ; Retina/metabolism ; Retina/pathology ; Retinoids/metabolism ; Stargardt Disease/genetics ; Stargardt Disease/therapy
Chemical Substances	A2-E (N-retinylidene-N-retinylethanolamine) ; ATP-Binding Cassette Transporters ; Abca4 protein, mouse ; Retinoids
Language	English
Publishing date	2019-09-30
Publishing country	United States
Document type	Journal Article ; Research Support, N.I.H., Extramural
ZDB-ID	1028152-6
ISSN	1557-7422 ; 1043-0342
ISSN (online)	1557-7422
ISSN	1043-0342
DOI	10.1089/hum.2019.132
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Article ; Online: Transgene expression of Stanniocalcin-1 provides sustained intraocular pressure reduction by increasing outflow facility.

Roddy, Gavin W / Roy Chowdhury, Uttio / Anderson, Kjersten J / Rinkoski, Tommy A / Hann, Cheryl R / Chiodo, Vince A / Smith, W Clay / Fautsch, Michael P

PloS one

2022 Volume 17, Issue 5, Page(s) e0269261

Abstract: Glaucoma is the leading cause of irreversible blindness worldwide. Therapies for glaucoma are directed toward reducing intraocular pressure (IOP), the leading risk factor and only reliable therapeutic target via topical medications or with procedural ... ...

Abstract	Glaucoma is the leading cause of irreversible blindness worldwide. Therapies for glaucoma are directed toward reducing intraocular pressure (IOP), the leading risk factor and only reliable therapeutic target via topical medications or with procedural intervention including laser or surgery. Though topical therapeutics are typically first line, less than 50% of patients take drops as prescribed. Sustained release technologies that decrease IOP for extended periods of time are being examined for clinical use. We recently identified Stanniocalcin-1, a naturally occurring hormone, as an IOP-lowering agent. Here, we show that a single injection into the anterior chamber of mice with an adeno-associated viral vector containing the transgene of stanniocalcin-1 results in diffuse and sustained expression of the protein and produces IOP reduction for up to 6 months. As the treatment effect begins to wane, IOP-lowering can be rescued with a repeat injection. Aqueous humor dynamic studies revealed an increase in outflow facility as the mechanism of action. This first-in-class therapeutic approach has the potential to improve care and reduce the rates of vision loss in the 80 million people worldwide currently affected by glaucoma.
MeSH term(s)	Animals ; Glaucoma/drug therapy ; Glaucoma/genetics ; Glycoproteins ; Humans ; Intraocular Pressure ; Mice ; Ocular Hypotension ; Tonometry, Ocular ; Transgenes
Chemical Substances	Glycoproteins ; teleocalcin (76687-96-2)
Language	English
Publishing date	2022-05-31
Publishing country	United States
Document type	Journal Article ; Research Support, Non-U.S. Gov't ; Research Support, N.I.H., Extramural
ZDB-ID	2267670-3
ISSN	1932-6203 ; 1932-6203
ISSN (online)	1932-6203
ISSN	1932-6203
DOI	10.1371/journal.pone.0269261
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Article ; Online: Neuroprotective Gene Therapy by Overexpression of the Transcription Factor MAX in Rat Models of Glaucomatous Neurodegeneration.

Lani-Louzada, Rafael / Marra, Camila / Dias, Mariana Santana / de Araújo, Victor Guedes / Abreu, Carla Andreia / Ribas, Vinícius Toledo / Adesse, Daniel / Allodi, Silvana / Chiodo, Vince / Hauswirth, William / Petrs-Silva, Hilda / Linden, Rafael

Investigative ophthalmology & visual science

2022 Volume 63, Issue 2, Page(s) 5

Abstract: Purpose: Based on our preview evidence that reduced nuclear content of the transcription factor Myc-associated protein X (MAX) is an early event associated with degeneration of retinal ganglion cells (RGCs), in the present study, our purpose was to test ...

Abstract	Purpose: Based on our preview evidence that reduced nuclear content of the transcription factor Myc-associated protein X (MAX) is an early event associated with degeneration of retinal ganglion cells (RGCs), in the present study, our purpose was to test whether the overexpression of human MAX had a neuroprotective effect against RGC injury. Methods: Overexpression of either MAX or green fluorescent protein (GFP) in the retina was achieved by intravitreal injections of recombinant adenovirus-associated viruses (rAAVs). Lister Hooded rats were used in three models of RGC degeneration: (1) cultures of retinal explants for 30 hours ex vivo from the eyes of 14-day-old rats that had received intravitreal injections of rAAV2-MAX or the control vector rAAV2-GFP at birth; (2) an optic nerve crush model, in which 1-month-old rats received intravitreal injection of either rAAV2-MAX or rAAV2-GFP and, 4 weeks later, were operated on; and (3) an ocular hypertension (OHT) glaucoma model, in which 1-month-old rats received intravitreal injection of either rAAV2-MAX or rAAV2-GFP and, 4 weeks later, were subject to cauterization of the limbal plexus. Cell death was estimated by detection of pyknotic nuclei and TUNEL technique and correlated with MAX immunocontent in an ex vivo model of retinal explants. MAX expression was detected by quantitative RT-PCR. In the OHT model, survival of RGCs was quantified by retrograde labeling with DiI or immunostaining for BRN3a at 14 days after in vivo injury. Functional integrity of RGCs was analyzed through pattern electroretinography, and damage to the optic nerve was examined in semithin sections. Results: In all three models of RGC insult, gene therapy by overexpression of MAX prevented RGC death. Also, ON degeneration and electrophysiologic deficits were prevented in the OHT model. Conclusions: Our experiments offer proof of concept for a novel neuroprotective gene therapy for glaucomatous neurodegeneration based on overexpression of MAX.
MeSH term(s)	Animals ; Basic Helix-Loop-Helix Leucine Zipper Transcription Factors/biosynthesis ; Basic Helix-Loop-Helix Leucine Zipper Transcription Factors/genetics ; Cell Death ; Disease Models, Animal ; Female ; Gene Expression Regulation ; Genetic Therapy/methods ; Glaucoma/complications ; Glaucoma/genetics ; Glaucoma/pathology ; Male ; Nerve Regeneration/genetics ; Neurodegenerative Diseases/etiology ; Neurodegenerative Diseases/genetics ; Neurodegenerative Diseases/therapy ; Neuroprotection/genetics ; Rats ; Retinal Ganglion Cells/metabolism ; Retinal Ganglion Cells/pathology
Chemical Substances	Basic Helix-Loop-Helix Leucine Zipper Transcription Factors ; Max protein, rat
Language	English
Publishing date	2022-02-01
Publishing country	United States
Document type	Journal Article ; Research Support, Non-U.S. Gov't
ZDB-ID	391794-0
ISSN	1552-5783 ; 0146-0404
ISSN (online)	1552-5783
ISSN	0146-0404
DOI	10.1167/iovs.63.2.5
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Article ; Online: Prdm1 overexpression causes a photoreceptor fate-shift in nascent, but not mature, bipolar cells.

Goodson, Noah B / Park, Ko U / Silver, Jason S / Chiodo, Vince A / Hauswirth, William W / Brzezinski, Joseph A

Developmental biology

2020 Volume 464, Issue 2, Page(s) 111–123

Abstract	The transcription factors Prdm1 (Blimp1) and Vsx2 (Chx10) work downstream of Otx2 to regulate photoreceptor and bipolar cell fates in the developing retina. Mice that lack Vsx2 fail to form bipolar cells while Prdm1 mutants form excess bipolars at the direct expense of photoreceptors. Excess bipolars in Prdm1 mutants appear to derive from rods, suggesting that photoreceptor fate remains mutable for some time after cells become specified. Here we tested whether bipolar cell fate is also plastic during development. To do this, we created a system to conditionally misexpress Prdm1 at different stages of bipolar cell development. We found that Prdm1 blocks bipolar cell formation if expressed before the fate choice decision occurred. When we misexpressed Prdm1 just after the decision to become a bipolar cell was made, some cells were reprogrammed into photoreceptors. In contrast, Prdm1 misexpression in mature bipolar cells did not affect cell fate. We also provide evidence that sustained misexpression of Prdm1 was selectively toxic to photoreceptors. Our data show that bipolar fate is malleable, but only for a short temporal window following fate specification. Prdm1 and Vsx2 act by stabilizing photoreceptor and bipolar fates in developing OTX2+ cells of the retina.
MeSH term(s)	Animals ; Cellular Reprogramming ; Gene Expression Regulation, Developmental ; Homeodomain Proteins/genetics ; Homeodomain Proteins/metabolism ; Mice ; Mice, Transgenic ; Mutation ; Otx Transcription Factors/genetics ; Otx Transcription Factors/metabolism ; Photoreceptor Cells, Vertebrate/cytology ; Photoreceptor Cells, Vertebrate/metabolism ; Positive Regulatory Domain I-Binding Factor 1/biosynthesis ; Positive Regulatory Domain I-Binding Factor 1/genetics ; Transcription Factors/genetics ; Transcription Factors/metabolism
Chemical Substances	Homeodomain Proteins ; Otx Transcription Factors ; Otx2 protein, mouse ; Prdm1 protein, mouse ; Transcription Factors ; Vsx2 protein, mouse ; Positive Regulatory Domain I-Binding Factor 1 (EC 2.1.1.-)
Language	English
Publishing date	2020-06-17
Publishing country	United States
Document type	Journal Article ; Research Support, N.I.H., Extramural ; Research Support, Non-U.S. Gov't
ZDB-ID	1114-9
ISSN	1095-564X ; 0012-1606
ISSN (online)	1095-564X
ISSN	0012-1606
DOI	10.1016/j.ydbio.2020.06.003
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Article ; Online: Rescue of cone function in cone-only

Hanke-Gogokhia, Christin / Chiodo, Vince A / Hauswirth, William W / Frederick, Jeanne M / Baehr, Wolfgang

Molecular vision

2018 Volume 24, Page(s) 834–846

Abstract: Purpose: Recessive mutations in the human : Methods: Nphp5: Results: In the : Conclusions: ... ...

Abstract	Purpose: Recessive mutations in the human Methods: Nphp5 Results: In the Conclusions: Nphp5
MeSH term(s)	Adenoviridae/genetics ; Adenoviridae/metabolism ; Amino Acid Sequence ; Animals ; Axoneme/metabolism ; Axoneme/ultrastructure ; Basic-Leucine Zipper Transcription Factors/deficiency ; Basic-Leucine Zipper Transcription Factors/genetics ; Calmodulin-Binding Proteins/deficiency ; Calmodulin-Binding Proteins/genetics ; Cilia/metabolism ; Cilia/ultrastructure ; Crosses, Genetic ; Cyclic Nucleotide Phosphodiesterases, Type 6/genetics ; Cyclic Nucleotide Phosphodiesterases, Type 6/metabolism ; Disease Models, Animal ; Eye Proteins/genetics ; Eye Proteins/metabolism ; Female ; G-Protein-Coupled Receptor Kinase 1/genetics ; G-Protein-Coupled Receptor Kinase 1/metabolism ; GTP-Binding Protein alpha Subunits/genetics ; GTP-Binding Protein alpha Subunits/metabolism ; Gene Expression Regulation ; Genetic Therapy/methods ; Genetic Vectors/chemistry ; Genetic Vectors/metabolism ; Humans ; Leber Congenital Amaurosis/genetics ; Leber Congenital Amaurosis/metabolism ; Leber Congenital Amaurosis/pathology ; Leber Congenital Amaurosis/therapy ; Male ; Mice ; Mice, Knockout ; Microtubule-Associated Proteins/genetics ; Microtubule-Associated Proteins/metabolism ; Phenotype ; Retinal Cone Photoreceptor Cells/metabolism ; Retinal Cone Photoreceptor Cells/pathology ; Sequence Alignment ; Sequence Homology, Amino Acid ; Transducin/genetics ; Transducin/metabolism
Chemical Substances	Basic-Leucine Zipper Transcription Factors ; Calmodulin-Binding Proteins ; Eye Proteins ; GTP-Binding Protein alpha Subunits ; Gnat1 protein, mouse ; IQCB1 protein, mouse ; Microtubule-Associated Proteins ; Nrl protein, mouse ; RP1 protein, mouse ; G-Protein-Coupled Receptor Kinase 1 (EC 2.7.11.14) ; Grk1 protein, mouse (EC 2.7.11.14) ; Cyclic Nucleotide Phosphodiesterases, Type 6 (EC 3.1.4.35) ; Pde6a protein, mouse (EC 3.1.4.35) ; Transducin (EC 3.6.5.1)
Language	English
Publishing date	2018-12-30
Publishing country	United States
Document type	Journal Article ; Research Support, N.I.H., Extramural ; Research Support, Non-U.S. Gov't
ZDB-ID	2017540-1
ISSN	1090-0535 ; 1090-0535
ISSN (online)	1090-0535
ISSN	1090-0535
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Article: Cone Phosphodiesterase-6γ' Subunit Augments Cone PDE6 Holoenzyme Assembly and Stability in a Mouse Model Lacking Both Rod and Cone PDE6 Catalytic Subunits.

Deng, Wen-Tao / Kolandaivelu, Saravanan / Dinculescu, Astra / Li, Jie / Zhu, Ping / Chiodo, Vince A / Ramamurthy, Visvanathan / Hauswirth, William W

Frontiers in molecular neuroscience

2018 Volume 11, Page(s) 233

Abstract: Rod and cone phosphodiesterase 6 (PDE6) are key effector enzymes of the vertebrate phototransduction pathway. Rod PDE6 consists of two catalytic subunits PDE6α and PDE6β and two identical inhibitory PDE6γ subunits, while cone PDE6 is composed of two ... ...

Abstract	Rod and cone phosphodiesterase 6 (PDE6) are key effector enzymes of the vertebrate phototransduction pathway. Rod PDE6 consists of two catalytic subunits PDE6α and PDE6β and two identical inhibitory PDE6γ subunits, while cone PDE6 is composed of two identical PDE6α' catalytic subunits and two identical cone-specific PDE6γ' inhibitory subunits. Despite their prominent function in regulating cGMP levels and therefore rod and cone light response properties, it is not known how each subunit contributes to the functional differences between rods and cones. In this study, we generated an
Language	English
Publishing date	2018-07-09
Publishing country	Switzerland
Document type	Journal Article
ZDB-ID	2452967-9
ISSN	1662-5099
ISSN	1662-5099
DOI	10.3389/fnmol.2018.00233
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Article ; Online: Human L- and M-opsins restore M-cone function in a mouse model for human blue cone monochromacy.

Deng, Wen-Tao / Li, Jie / Zhu, Ping / Chiodo, Vince A / Smith, W Clay / Freedman, Beau / Baehr, Wolfgang / Pang, Jijing / Hauswirth, William W

Molecular vision

2018 Volume 24, Page(s) 17–28

Abstract: Purpose: Blue cone monochromacy (BCM) is an X-linked congenital vision disorder characterized by complete loss or severely reduced L- and M-cone function. Patients with BCM display poor visual acuity, severely impaired color discrimination, myopia, ... ...

Abstract	Purpose: Blue cone monochromacy (BCM) is an X-linked congenital vision disorder characterized by complete loss or severely reduced L- and M-cone function. Patients with BCM display poor visual acuity, severely impaired color discrimination, myopia, nystagmus, and minimally detectable cone-mediated electroretinogram. Recent studies of patients with BCM with adaptive optics scanning laser ophthalmoscopy (AOSLO) showed that they have a disrupted cone mosaic with reduced numbers of cones in the fovea that is normally dominated by L- and M-cones. The remaining cones in the fovea have significantly shortened outer segments but retain sufficient structural integrity to serve as potential gene therapy targets. In this study, we tested whether exogenously expressed human L- and M-opsins can rescue M-cone function in an M-opsin knockout ( Methods: Adeno-associated virus type 5 (AAV5) vectors expressing OPN1LW, OPN1MW, or C-terminal tagged OPN1LW-Myc, or OPN1MW-HA driven by a cone-specific promoter were injected subretinally into one eye of Results: We showed that cones in the dorsal retina of the Conclusions: Exogenously expressed human opsins can regenerate cone outer segments and rescue M-cone function in
MeSH term(s)	Animals ; Color Vision Defects/genetics ; Color Vision Defects/metabolism ; Color Vision Defects/pathology ; Color Vision Defects/therapy ; Dependovirus/genetics ; Dependovirus/metabolism ; Disease Models, Animal ; Fovea Centralis/metabolism ; Fovea Centralis/pathology ; Gene Expression ; Genetic Complementation Test ; Genetic Therapy/methods ; Genetic Vectors/chemistry ; Genetic Vectors/metabolism ; Humans ; Mice ; Mice, Knockout ; Ophthalmoscopy ; Promoter Regions, Genetic ; Retinal Photoreceptor Cell Outer Segment/metabolism ; Retinal Photoreceptor Cell Outer Segment/pathology ; Rod Opsins/genetics ; Rod Opsins/metabolism ; Transgenes
Chemical Substances	Rod Opsins ; long-wavelength opsin ; middle-wavelength opsin
Language	English
Publishing date	2018-01-08
Publishing country	United States
Document type	Journal Article ; Research Support, N.I.H., Extramural ; Research Support, Non-U.S. Gov't
ZDB-ID	2017540-1
ISSN	1090-0535 ; 1090-0535
ISSN (online)	1090-0535
ISSN	1090-0535
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