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1. WO2016149664 - METHODS AND COMPOSITIONS FOR RESTORATION OF CONE FUNCTION IN BCM

Publication Number WO/2016/149664
Publication Date 22.09.2016
International Application No. PCT/US2016/023252
International Filing Date 18.03.2016
IPC
A61K 39/12 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
39Medicinal preparations containing antigens or antibodies
12Viral antigens
A61K 48/00 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
C12N 15/00 2006.01
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
C07H 21/04 2006.01
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
21Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
04with deoxyribosyl as saccharide radical
CPC
A61K 38/177
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38Medicinal preparations containing peptides
16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
17from animals; from humans
177Receptors; Cell surface antigens; Cell surface determinants
A61K 48/0058
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
005characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
A61K 48/0075
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
0075characterised by an aspect of the delivery route, e.g. oral, subcutaneous
A61K 9/0048
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
9Medicinal preparations characterised by special physical form
0012Galenical forms characterised by the site of application
0048Eye, e.g. artificial tears
A61P 27/02
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
27Drugs for disorders of the senses
02Ophthalmic agents
C07K 14/723
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435from animals; from humans
705Receptors; Cell surface antigens; Cell surface determinants
72for hormones
723G protein coupled receptor, e.g. TSHR-thyrotropin-receptor, LH/hCG receptor, FSH receptor
Applicants
  • UNIVERSITY OF FLORIDA RESEARCH FOUNDATION, INC. [US]/[US]
Inventors
  • PANG, Ji-Jing
  • HAUSWIRTH, William, W.
Agents
  • WALLER, Patrick, R.H.
Priority Data
62/134,82218.03.2015US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) METHODS AND COMPOSITIONS FOR RESTORATION OF CONE FUNCTION IN BCM
(FR) MÉTHODES ET COMPOSITIONS VISANT À RÉTABLIR LE FONCTIONNEMENT DES CÔNES DANS LE MONOCHROMATISME À CÔNES BLEUS
Abstract
(EN)
Provided are nucleic acid sequences, recombinant adeno-associated viral particles, compositions, and methods related to treating cone monochromacies, such as blue cone monochromacy (BCM). Specifically, the nucleic acid sequences, recombinant adeno-associated viral particles, compositions, and methods involve use of an M-opsin gene operably linked to a cone-specific promoter. Further disclosed is the sequence of a cone-specific PR2.1 promoter.
(FR)
L'invention concerne des acides nucléiques, des particules virales adéno-associées recombinées, des compositions et des méthodes associées au traitement des monochromatismes, tels que le monochromatisme à cônes bleus. Dans certains exemples, les acides nucléiques, les particules virales adéno-associées recombinées, les compositions et les méthodes impliquent l'utilisation d'un gène de l'opsine M fonctionnellement lié à un promoteur spécifique des cônes.
Also published as
Latest bibliographic data on file with the International Bureau