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1. (WO2019046069) ADENO-ASSOCIATED VIRUS CAPSID VARIANTS AND METHODS OF USE THEREOF
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Pub. No.: WO/2019/046069 International Application No.: PCT/US2018/047561
Publication Date: 07.03.2019 International Filing Date: 22.08.2018
IPC:
C12N 7/00 (2006.01) ,A61K 48/00 (2006.01) ,G01N 33/50 (2006.01)
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
7
Viruses, e.g. bacteriophages; Compositions thereof; Preparation or purification thereof
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48
Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
G PHYSICS
01
MEASURING; TESTING
N
INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
33
Investigating or analysing materials by specific methods not covered by groups G01N1/-G01N31/131
48
Biological material, e.g. blood, urine; Haemocytometers
50
Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
Applicants:
THE REGENTS OF THE UNIVERSITY OF CALIFORNIA [US/US]; 1111 Franklin Street, 12th Floor Oakland, California 94607, US
Inventors:
SCHAFFER, David V.; US
OJALA, David Stephen; US
ROMERO, Philip A.; US
Agent:
BORDEN, Paula A.; US
Priority Data:
62/551,13328.08.2017US
Title (EN) ADENO-ASSOCIATED VIRUS CAPSID VARIANTS AND METHODS OF USE THEREOF
(FR) VARIANTS DE CAPSIDE DE VIRUS ADÉNO-ASSOCIÉ ET LEURS PROCÉDÉS D’UTILISATION
Abstract:
(EN) The present disclosure provides recombinant adeno-associated virus virions with variant capsid protein, where the recombinant AAV (rAAV) virions exhibit one or more of increased ability to cross neuronal cell barriers, increased infectivity of a neural stem cell, increased infectivity of a neuronal cell, and reduced susceptibility to antibody neutralization, compared to a control AAV, and where the rAAV virions comprise a heterologous nucleic acid. The present disclosure provides methods of delivering a gene product to a neural stem cell or a neuronal cell in an individual. The present disclosure also provides methods of modifying a target nucleic acid present in a neural stem cell or neuronal cell.
(FR) La présente invention concerne des virions de virus recombinant adéno-associé à une protéine de capside variante, où les virions AAV recombinants (rAAV) font preuve d’une ou plusieurs capacités accrues de traverser les barrières des cellules neuronales, d'infectivité accrue d’une cellule souche neuronale, d'infectivité accrue d’une cellule neuronale, et de sensibilité réduite à la neutralisation d’anticorps, comparés à un AAV témoin, et où les virions rAAV comprennent un acide nucléique hétérologue. La présente invention concerne des procédés d’administration d’un produit génique à une cellule souche neuronale ou une cellule neuronale chez un individu. La présente invention concerne également des procédés de modification d’un acide nucléique cible présent dans une cellule souche neuronale ou une cellule neuronale.
Designated States: AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JO, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW
African Regional Intellectual Property Organization (ARIPO) (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW)
Eurasian Patent Office (AM, AZ, BY, KG, KZ, RU, TJ, TM)
European Patent Office (EPO) (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR)
African Intellectual Property Organization (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG)
Publication Language: English (EN)
Filing Language: English (EN)