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1. WO2020112967 - MODULATION OF SPTLC1 VIA RECOMBINANT ADENO-ASSOCIATED VECTORS

Publication Number WO/2020/112967
Publication Date 04.06.2020
International Application No. PCT/US2019/063558
International Filing Date 27.11.2019
IPC
C12N 15/113 2010.01
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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
09Recombinant DNA-technology
11DNA or RNA fragments; Modified forms thereof
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
C12N 15/86 2006.01
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12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79Vectors or expression systems specially adapted for eukaryotic hosts
85for animal cells
86Viral vectors
A61K 35/76 2015.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
35Medicinal preparations containing materials or reaction products thereof with undetermined constitution
66Microorganisms or materials therefrom
76Viruses; Subviral particles; Bacteriophages
A61P 25/00 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
25Drugs for disorders of the nervous system
CPC
C12N 15/1137
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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
09Recombinant DNA-technology
11DNA or RNA fragments; Modified forms thereof
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
1137against enzymes
C12N 2310/141
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12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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2310Structure or type of the nucleic acid
10Type of nucleic acid
14interfering N.A.
141MicroRNAs, miRNAs
C12N 2310/531
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12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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2310Structure or type of the nucleic acid
50Physical structure
53partially self-complementary or closed
531Stem-loop; Hairpin
C12N 2750/14143
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12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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2750ssDNA Viruses
00011ssDNA Viruses
14011Parvoviridae
14111Dependovirus, e.g. adenoassociated viruses
14141Use of virus, viral particle or viral elements as a vector
14143viral genome or elements thereof as genetic vector
C12Y 203/0105
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
YENZYMES
203Acyltransferases (2.3)
01transferring groups other than amino-acyl groups (2.3.1)
0105Serine C-palmitoyltransferase (2.3.1.50)
Applicants
  • UNIVERSITY OF MASSACHUSETTS [US]/[US]
Inventors
  • MUELLER, Christian
  • CABRERA, Gabriela, Toro
  • BROWN, Robert, H., Jr.
Agents
  • BEYERSDORF, Matthew, S.
  • ATTISHA, Michael, J.
  • AMUNDSEN, Eric
  • BAKER, C., Hunter
  • ALAM, Saad
Priority Data
62/772,66829.11.2018US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) MODULATION OF SPTLC1 VIA RECOMBINANT ADENO-ASSOCIATED VECTORS
(FR) MODULATION DE SPTLC1 PAR L'INTERMÉDIAIRE DE VECTEURS ADÉNO-ASSOCIÉS RECOMBINÉS
Abstract
(EN)
The disclosure provides, in some aspects, compositions (e.g., isolated nucleic acids and rAAVs) comprising a transgene which encodes at least one inhibitory nucleic acid which decreases expression of a gene encoding a serine palmitoyltransferase protein (e.g., SPTLC1). In some aspects, the disclosure relates to methods of treating hereditary sensory and autonomic neuropathy 1 (HSAN1) by administering the compositions described by the disclosure to a cell or subject.
(FR)
L'invention concerne, dans certains aspects, des compositions (par exemple, des acides nucléiques isolés et des rAAV) comprenant un transgène codant au moins un acide nucléique inhibiteur qui diminue l'expression d'un gène codant pour une protéine de sérine palmitoyltransférase (par exemple SPTLC1). Dans certains aspects, l'invention concerne des procédés de traitement de la neuropathie héréditaire sensitive et autonomique type 1 (HSAN1) par l'administration des compositions décrites par l'invention à une cellule ou à un sujet.
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