MX2021003303A - Composiciones y metodos para restaurar la expresion del gen ube3a paterno en sindrome de angelman humano. - Google Patents

Composiciones y metodos para restaurar la expresion del gen ube3a paterno en sindrome de angelman humano.

Info

Publication number
MX2021003303A
MX2021003303A MX2021003303A MX2021003303A MX2021003303A MX 2021003303 A MX2021003303 A MX 2021003303A MX 2021003303 A MX2021003303 A MX 2021003303A MX 2021003303 A MX2021003303 A MX 2021003303A MX 2021003303 A MX2021003303 A MX 2021003303A
Authority
MX
Mexico
Prior art keywords
compositions
human
methods
expression
restore
Prior art date
Application number
MX2021003303A
Other languages
English (en)
Spanish (es)
Inventor
Stormy Chamberlain
Noelle Germain
Original Assignee
Univ Connecticut
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Univ Connecticut filed Critical Univ Connecticut
Publication of MX2021003303A publication Critical patent/MX2021003303A/es

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
    • C12N15/1137—Non-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 against enzymes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/121—Hammerhead
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/122—Hairpin
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/128—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes processing or releasing ribozyme
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15042—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/007—Vectors comprising a special translation-regulating system cell or tissue specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
MX2021003303A 2018-09-21 2019-09-20 Composiciones y metodos para restaurar la expresion del gen ube3a paterno en sindrome de angelman humano. MX2021003303A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862734435P 2018-09-21 2018-09-21
PCT/US2019/052272 WO2020061528A1 (en) 2018-09-21 2019-09-20 Compositions and methods to restore paternal ube3a gene expression in human angelman syndrome

Publications (1)

Publication Number Publication Date
MX2021003303A true MX2021003303A (es) 2021-06-23

Family

ID=69887937

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2021003303A MX2021003303A (es) 2018-09-21 2019-09-20 Composiciones y metodos para restaurar la expresion del gen ube3a paterno en sindrome de angelman humano.

Country Status (10)

Country Link
US (2) US12221609B2 (https=)
EP (1) EP3833398A4 (https=)
JP (1) JP7546295B2 (https=)
KR (1) KR102782761B1 (https=)
CN (2) CN119592561A (https=)
AU (2) AU2019344006B2 (https=)
CA (1) CA3113648A1 (https=)
IL (1) IL281563B1 (https=)
MX (1) MX2021003303A (https=)
WO (1) WO2020061528A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3113648A1 (en) 2018-09-21 2020-03-26 University Of Connecticut Compositions and methods to restore paternal ube3a gene expression in human angelman syndrome
EP4142802A4 (en) * 2020-04-28 2024-07-24 The Trustees of The University of Pennsylvania COMPOSITIONS AND USES THEREOF FOR THE TREATMENT OF ANGELMAN SYNDROME
US20240318198A1 (en) * 2021-02-03 2024-09-26 The Board Of Regents Of The University Of Texas System Gene therapy for angelman syndrome
IL315359A (en) * 2022-03-07 2024-11-01 Univ Connecticut shRNA TARGETING SNORD115 LOCATIONS TO RESTORE PATERNAL UBE3A GENE EXPRESSION IN ANGELMAN SYNDROME
KR20230152608A (ko) 2022-04-27 2023-11-03 중앙대학교 산학협력단 CRISPR-Cas12f1 시스템을 이용한 유전체의 단일 뉴클레오타이드 편집 방법 및 이의 용도

Family Cites Families (40)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
FR2705686B1 (fr) 1993-05-28 1995-08-18 Transgene Sa Nouveaux adénovirus défectifs et lignées de complémentation correspondantes.
FR2711523B1 (fr) 1993-10-26 1996-02-16 Transgene Sa Procédé de préparation d'un aérosol viral.
WO1995022618A1 (en) 1994-02-22 1995-08-24 Dana-Farber Cancer Institute Nucleic acid delivery system, method of synthesis and uses thereof
US5837520A (en) 1995-03-07 1998-11-17 Canji, Inc. Method of purification of viral vectors
FR2737222B1 (fr) 1995-07-24 1997-10-17 Transgene Sa Nouveaux vecteurs viraux et lignee pour la therapie genique
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
EP0944717A1 (fr) 1996-07-01 1999-09-29 Aventis Pharma S.A. Procede de production d'adenovirus recombinants
ES2278399T3 (es) 1996-11-20 2007-08-01 Introgen Therapeutics, Inc. Metodo mejorado para la produccion y purificacion de vectores adenovirales.
ATE477320T1 (de) 1996-12-13 2010-08-15 Schering Corp Methoden zur virus-reinigung
FR2763958A1 (fr) 1997-05-29 1998-12-04 Transgene Sa Produit de combinaison associant un acide nucleique a une substance desorganisant la matrice extracellulaire pour la therapie genique
CA2242035C (en) 1997-07-01 2004-11-23 Transgene S.A. Compositions useful for transferring therapeutically active substances into a target cell, and their use in gene therapy
JP2002513775A (ja) 1998-05-06 2002-05-14 トランジェーヌ、ソシエテ、アノニム 細胞の中へのポリヌクレオチドのトランスフェクションを改良する治療組成物を調製するためのヌクレアーゼインヒビターまたはインターロイキン−１０（ｉｌ−１０）の使用、および遺伝子治療に有用な組成物
JP3864610B2 (ja) 1998-05-21 2007-01-10 旭硝子株式会社 水分散型撥水撥油剤組成物およびその製造方法
EP0998945A1 (en) 1998-09-30 2000-05-10 Transgene S.A. Use of magnesium (Mg2+) for the enhancement of gene delivery in gene therapy
CA2356373A1 (fr) 1998-12-31 2000-07-13 Aventis Pharma S.A. Methode de separation de particules virales
EP1155120B1 (fr) 1999-02-22 2006-07-05 Transgene S.A. Procede d'obtention d'une preparation virale purifiee
JP2003518511A (ja) 1999-12-28 2003-06-10 トランジェーヌ、ソシエテ、アノニム ポリヌクレオチドを細胞へトランスフェクションするための組成物を調製するためのリチウム（Ｌｉ＋）の使用および遺伝子治療で有用な組成物
CA2500224C (en) 2002-09-25 2015-04-28 University Of Massachusetts In vivo gene silencing by chemically modified and stable sirna
CA2519860C (en) 2003-03-21 2018-01-16 Santaris Pharma A/S Short interfering rna (sirna) analogues
DK1648931T3 (da) 2003-07-21 2011-03-07 Transgene Sa Multifunktionelle cytokiner
WO2005073378A1 (en) 2004-01-30 2005-08-11 Santaris Pharma A/S MODIFIED SHORT INTERFERING RNA (MODIFIED siRNA)
US7361468B2 (en) * 2004-07-02 2008-04-22 Affymetrix, Inc. Methods for genotyping polymorphisms in humans
US20080213891A1 (en) 2004-07-21 2008-09-04 Alnylam Pharmaceuticals, Inc. RNAi Agents Comprising Universal Nucleobases
US8101741B2 (en) 2005-11-02 2012-01-24 Protiva Biotherapeutics, Inc. Modified siRNA molecules and uses thereof
WO2007124452A2 (en) 2006-04-20 2007-11-01 The Regents Of The University Of California Methods for expressing multiple sirna and shrna from a single vector
US8680064B2 (en) 2008-09-15 2014-03-25 The Board Of Trustees Of The Leland Stanford Junior University ShRNA gene therapy for treatment of ischemic heart disease
WO2011111072A2 (en) 2010-03-10 2011-09-15 Transgene Biotek Ltd. Adeno-associated virus ezh2 shrna (aav ezh2 shrna)therapy for breast cancer
US9163236B2 (en) 2010-12-06 2015-10-20 The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services Pharmaceutical composition comprising NANOG SHRNA, and method of using NANOG SHRNA to treat cancer
CA2863964C (en) * 2012-02-07 2021-10-26 Global Bio Therapeutics Usa, Inc. Compartmentalized method of nucleic acid delivery and compositions and uses thereof
US20130259927A1 (en) 2012-04-02 2013-10-03 Gradalis, Inc. Ewing's Sarcoma Bifunctional shRNA Design
ES2809199T3 (es) 2012-06-25 2021-03-03 Ionis Pharmaceuticals Inc Modulación de la expresión de UBE3A-ATS
EP2872183B1 (en) 2012-07-11 2018-09-26 The Trustees Of The University Of Pennsylvania Aav-mediated gene therapy for rpgr x-linked retinal degeneration
US10233495B2 (en) 2012-09-27 2019-03-19 The Hospital For Sick Children Methods and compositions for screening and treating developmental disorders
KR20160089528A (ko) * 2013-12-12 2016-07-27 더 브리검 앤드 우먼즈 하스피털, 인크. 신경퇴행성 질환의 치료
WO2016086104A1 (en) * 2014-11-25 2016-06-02 Ionis Pharmaceuticals, Inc. Modulation of ube3a-ats expression
HUE067905T2 (hu) * 2015-11-12 2024-11-28 Hoffmann La Roche Oligonukleotidok paternális UBE3A expresszió indukálására
EP3448875A4 (en) 2016-04-29 2020-04-08 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
WO2020051417A2 (en) 2018-09-07 2020-03-12 The Chemours Company Fc, Llc Fluorine removal from antimony fluorohalide catalyst using chlorocarbons
CA3113648A1 (en) 2018-09-21 2020-03-26 University Of Connecticut Compositions and methods to restore paternal ube3a gene expression in human angelman syndrome

Also Published As

Publication number Publication date
AU2019344006B2 (en) 2025-11-27
KR20210065125A (ko) 2021-06-03
KR102782761B1 (ko) 2025-03-14
WO2020061528A1 (en) 2020-03-26
US20250136994A1 (en) 2025-05-01
US12221609B2 (en) 2025-02-11
AU2019344006A1 (en) 2021-03-25
EP3833398A1 (en) 2021-06-16
EP3833398A4 (en) 2021-12-22
CN113164624B (zh) 2024-09-24
CN113164624A (zh) 2021-07-23
CA3113648A1 (en) 2020-03-26
US20210332368A1 (en) 2021-10-28
IL281563B1 (en) 2026-01-01
AU2026200604A1 (en) 2026-02-19
JP2022501044A (ja) 2022-01-06
JP7546295B2 (ja) 2024-09-06
IL281563A (en) 2021-05-31
CN119592561A (zh) 2025-03-11

Similar Documents

Publication Publication Date Title
MX2021003303A (es) Composiciones y metodos para restaurar la expresion del gen ube3a paterno en sindrome de angelman humano.
CY1124845T1 (el) Μεθοδοι παροχης μονοκλωνου rna
CY1125373T1 (el) Λιπιδικα νανοσωματιδια για την παροχη τροποποιημενου rna που κωδικοποιει ενα πολυπεπτιδιο vegf-a
CY1122347T1 (el) 3' utr αλληλουχιες για τη σταθεροποιηση toy rna
CL2018001189A1 (es) Oligonucleótidos para inducir la expresión paterna de ube3a
CY1125364T1 (el) Συνθεσεις και μεθοδοι για την αναστολη της εκφρασης του γονιδιου ηαο1 (οξειδαση υδροξυοξεος 1 (γλυκολικη οξειδαση)
CY1121826T1 (el) Προφαρμακα καρβιντοπας και l-ντοπας και η χρηση τους στην αγωγη της νοσου parkinson
CY1121348T1 (el) Mrna θεραπεια για την θεραπευτικη αγωγη οφθαλμικων ασθενειων
BR112016029178A2 (pt) composições e métodos para a expressão de rnas guia de crispr usando o promotor h1
CY1123947T1 (el) Βελτιστοποιημενος υποκινητης rpe65 και βελτιστοποιημενες κωδικοποιητικες αλληλουχιες
MX390848B (es) Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos.
MX2019008380A (es) Composiciones de arni contra el componente c5 del complemento y metodos para su uso.
MX2020000676A (es) Composiciones y metodos para tratar beta-hemoglobinopatias.
MX2019015143A (es) Rna guias modificados, complejos crispr-ribonucleoproteina y metodos de uso.
MX359201B (es) Polipeptidos que se unen al complemento humano 5 (c5).
CR20150604A (es) Proceso para la preparación de eritrocitos cargados con una o más sustancias de interés farmacéutico y eritrocitos así obtenidos
MX2016004319A (es) Composiciones y metodos para inhibir la expresion del gen de sintasa de acido 5'-aminolevulinico 1 (alas1).
MX392293B (es) Composición química de ácido hialurónico
WO2019213128A8 (en) Compositions and therapeutic methods of microrna gene delivery
MX2020013667A (es) Uso de mir-92a o mir-145 en el tratamiento del sindrome de angelman.
PH12015502037B1 (en) Activating adiponectin by casein hydrolysate
MX2024010820A (es) Arnhc que se dirige a ubicaciones de snord115 para restaurar la expresión génica del ube3a paterno en el síndrome de angelman
MX2024010821A (es) Arnhc dirigido a ube3a-ats para restaurar la expresión del gen ube3a paterno en el síndrome angelman
BR112017002781A2 (pt) cassete de expressão aprimorado para acondicionamento e expressão de variantes do fator viii para o tratamento de distúrbios de hemostasia