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Compositions and methods for intravitreal delivery of polynucleotides to retinal cones

a technology of polynucleotide and retinal cone, which is applied in the field ofviral-based gene therapy of retinal disorders, can solve problems such as the risk of creating additional damage to retinal tissu

Pending Publication Date: 2021-12-16
UNIV OF WASHINGTON +1
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Benefits of technology

The approach enables robust and efficient gene expression in cone photoreceptors, particularly in the foveal region, enhancing the treatment of cone-associated disorders by achieving higher transduction efficiency and therapeutic gene delivery.

Problems solved by technology

However, these vectors must be administered by subretinal injection, a procedure that disrupts the structure of the retina and carries with it a risk of creating additional damage to retinal tissue that is often already damaged by the disorder being treated.

Method used

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  • Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
  • Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
  • Compositions and methods for intravitreal delivery of polynucleotides to retinal cones

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Background

[0174]New therapies are needed for the treatment of many cone photoreceptor associated disorders, including macular dystrophies such as cone-rod dystrophy, cone dystrophy, Stargardt macular dystrophy, and achromatopsia; color vision disorders such as protan, deutan, and tritan defects; and vision disorders of the central macula such as age-related macular degeneration, macular telangiectasia, retinitis pigmentosa, diabetic retinopathy, retinal vein occlusions, glaucoma, Sorsby's fundus dystrophy, adult vitelliform macular dystrophy, Best's disease, and X-linked retinoschisis. As these vision disorders are associated with a loss of function and / or viability of the cone photoreceptors, it is hypothesized that these disorders may be treatable by delivering a therapeutic gene to cone photoreceptors to rescue cone viability and function.

[0175]To that end, the polynucleotide cassette “pMNTC” was designed in which enhancer, promoter, 5′UTR, intron, Kozak, and polyadenylation sequ...

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Abstract

Methods and compositions are provided for intravitreally delivering a polynucleotide to cone photoreceptors. Aspects of the methods include injecting a recombinant adeno-associated virus comprising a polynucleotide of interest into the vitreous of the eye. These methods and compositions find particular use in treating ocular disorders associated with cone dysfunction and / or death.

Description

CROSS REFERENCE TO RELATED APPLICATIONS[0001]This application is a continuation application of U.S. patent application Ser. No. 15 / 554,664, filed Aug. 30, 2017, which is a U.S. National Phase Application of International Patent Application No. PCT / US2016 / 020482, filed Mar. 2, 2016; which claims the benefit under 35 U.S.C. § 119(e) of U.S. Provisional Application No. 62 / 127,194, filed on Mar. 2, 2015, and U.S. Provisional Application No. 62 / 134,466, filed on Mar. 17, 2015; each of which is incorporated by reference herein in its entirety.STATEMENT REGARDING SEQUENCE LISTING[0002]The Sequence Listing associated with this application is provided in text format in lieu of a paper copy, and is hereby incorporated by reference into the specification. The name of the text file containing the Sequence Listing is AVBI_006_04 US_ST25.txt. The text file is 74 KB, was created on May 5, 2021, and is being submitted electronically via EFS-Web.FIELD OF THE INVENTION[0003]This invention pertains to...

Claims

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Application Information

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Patent Type & Authority Applications(United States)
IPC IPC(8): C07K14/005A61K48/00C12N15/86C12N15/861
CPCC07K14/005A61K48/0066A61K48/0075C12N7/00C12N15/861C12N2750/14122C12N2750/14143C12N15/86A61P27/02C12N2750/14121
Inventor CHALBERG, JR., THOMAS W.NEITZ, JAYNEITZ, MAUREEN
Owner UNIV OF WASHINGTON
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