MX2019005235A - Constructos de expresion de frataxina. - Google Patents

Constructos de expresion de frataxina.

Info

Publication number
MX2019005235A
MX2019005235A MX2019005235A MX2019005235A MX2019005235A MX 2019005235 A MX2019005235 A MX 2019005235A MX 2019005235 A MX2019005235 A MX 2019005235A MX 2019005235 A MX2019005235 A MX 2019005235A MX 2019005235 A MX2019005235 A MX 2019005235A
Authority
MX
Mexico
Prior art keywords
expression constructs
frataxin expression
frataxin
ataxia
vectors
Prior art date
Application number
MX2019005235A
Other languages
English (en)
Inventor
Schauer Stephen
Thomas Darby
Robinson Gregory
Pykett Mark
Thorn Richard
Gruis Kirsten
Original Assignee
Intrexon Corp
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 Intrexon Corp filed Critical Intrexon Corp
Publication of MX2019005235A publication Critical patent/MX2019005235A/es

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • 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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
    • C12N2830/002—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Neurology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Immunology (AREA)
  • Toxicology (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)

Abstract

La invención proporciona polinucleótidos, vectores y virus que expresan frataxina y los métodos para tratar la Ataxia de Friedreich.
MX2019005235A 2016-11-09 2017-11-08 Constructos de expresion de frataxina. MX2019005235A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662419621P 2016-11-09 2016-11-09
PCT/US2017/060680 WO2018089527A1 (en) 2016-11-09 2017-11-08 Frataxin expression constructs

Publications (1)

Publication Number Publication Date
MX2019005235A true MX2019005235A (es) 2019-12-05

Family

ID=60629792

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2019005235A MX2019005235A (es) 2016-11-09 2017-11-08 Constructos de expresion de frataxina.

Country Status (14)

Country Link
US (3) US11116852B2 (es)
EP (1) EP3538660A1 (es)
CN (1) CN110139933B (es)
AU (1) AU2017359342B2 (es)
BR (1) BR112019009446A2 (es)
CA (1) CA3042706A1 (es)
CO (1) CO2019005611A2 (es)
EA (1) EA201990864A1 (es)
IL (1) IL266358A (es)
MX (1) MX2019005235A (es)
NZ (1) NZ752941A (es)
SA (1) SA519401756B1 (es)
WO (1) WO2018089527A1 (es)
ZA (1) ZA201902870B (es)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20210068068A (ko) * 2018-09-28 2021-06-08 보이저 테라퓨틱스, 인크. 조작된 프로모터를 갖는 프라탁신 발현 구축물 및 그의 사용 방법
WO2021030303A1 (en) * 2019-08-12 2021-02-18 Voyager Therapeutics, Inc. High-sensitivity immunoassay for the detection of frataxin in biofluids
JP2022548270A (ja) * 2019-09-13 2022-11-17 ラセルタ セラピューティクス， インコーポレイテッド フリードライヒ運動失調症の治療のための組成物及び方法
JP2023535632A (ja) 2020-07-27 2023-08-18 アンジャリウム バイオサイエンシズ エージー Ｄｎａ分子の組成物、その作製方法、及びその使用方法
CA3231881A1 (en) * 2021-09-17 2023-03-23 Dwaipayan Sen Frataxin gene therapy
WO2023235888A2 (en) * 2022-06-03 2023-12-07 Scribe Therapeutics Inc. COMPOSITIONS AND METHODS FOR CpG DEPLETION

Family Cites Families (84)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5117057A (en) 1985-10-21 1992-05-26 Rohm And Haas Company Insecticidal N' substituted-N-N'-disubstituted-hydrazines
US4985461A (en) 1985-10-21 1991-01-15 Rohm And Haas Company Insecticidal N'-substituted-N,N'-diacylhydrazines
FR2593630B1 (fr) 1986-01-27 1988-03-18 Maurice Francois Ecran d'affichage a matrice active a resistance de drain et procedes de fabrication de cet ecran
US4861719A (en) 1986-04-25 1989-08-29 Fred Hutchinson Cancer Research Center DNA constructs for retrovirus packaging cell lines
US5225443A (en) 1986-05-01 1993-07-06 Rohm And Haas Company Insecticidal N'-substituted-N'-substituted N,N'-diacylhydrazines
US4981784A (en) 1987-12-02 1991-01-01 The Salk Institute For Biological Studies Retinoic acid receptor method
US5278056A (en) 1988-02-05 1994-01-11 The Trustees Of Columbia University In The City Of New York Retroviral packaging cell lines and process of using same
US5354844A (en) 1989-03-16 1994-10-11 Boehringer Ingelheim International Gmbh Protein-polycation conjugates
US5703055A (en) 1989-03-21 1997-12-30 Wisconsin Alumni Research Foundation Generation of antibodies through lipid mediated DNA delivery
US5693622A (en) 1989-03-21 1997-12-02 Vical Incorporated Expression of exogenous polynucleotide sequences cardiac muscle of a mammal
US5670488A (en) 1992-12-03 1997-09-23 Genzyme Corporation Adenovirus vector for gene therapy
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
CA2043775A1 (en) 1990-06-14 1991-12-15 Dat P. Le Dibenzoylakylcyanohydrazines
IL100643A (en) 1991-01-25 1996-10-31 Nippon Kayaku Kk History of hydrazine and pesticides containing these histories as an active ingredient
US5530028A (en) 1992-11-23 1996-06-25 Rohm And Haas Company Insecticidal N'-substituted-N,N'-diacylhydrazines
US6013836A (en) 1992-02-28 2000-01-11 Rohm And Haas Company Insecticidal N'-substituted-N,N'-disubstitutedhydrazines
CA2156725A1 (en) 1993-02-22 1994-09-01 Warren S. Pear Production of high titer helper-free retroviruses by transient transfection
FR2712812B1 (fr) 1993-11-23 1996-02-09 Centre Nat Rech Scient Composition pour la production de produits thérapeutiques in vivo.
FR2714830B1 (fr) 1994-01-10 1996-03-22 Rhone Poulenc Rorer Sa Composition contenant des acides nucléiques, préparation et utilisations.
FR2715847B1 (fr) 1994-02-08 1996-04-12 Rhone Poulenc Rorer Sa Composition contenant des acides nucléiques, préparation et utilisations.
US6150137A (en) 1994-05-27 2000-11-21 Ariad Pharmaceuticals, Inc. Immunosuppressant target proteins
FR2727679B1 (fr) 1994-12-05 1997-01-03 Rhone Poulenc Rorer Sa Nouveaux agents de transfection et leurs applications pharmaceutiques
IL116816A (en) 1995-01-20 2003-05-29 Rhone Poulenc Rorer Sa Cell for the production of a defective recombinant adenovirus or an adeno-associated virus and the various uses thereof
FR2730637B1 (fr) 1995-02-17 1997-03-28 Rhone Poulenc Rorer Sa Composition pharmaceutique contenant des acides nucleiques, et ses utilisations
CA2213340C (en) 1995-03-03 2011-07-26 Novartis Ag Control of gene expression in plants by receptor mediated transactivation in the presence of a chemical ligand
JPH11506319A (ja) 1995-05-26 1999-06-08 ゼネカ・リミテッド エクジソンレセプターを含む遺伝子スイッチ
US6187757B1 (en) 1995-06-07 2001-02-13 Ariad Pharmaceuticals, Inc. Regulation of biological events using novel compounds
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
US6150091A (en) 1996-03-06 2000-11-21 Baylor College Of Medicine Direct molecular diagnosis of Friedreich ataxia
US6723531B2 (en) 1996-04-05 2004-04-20 The Salk Institute For Biological Studies Method for modulating expression of exogenous genes in mammalian systems, and products related thereto
US20020048792A1 (en) 1997-08-26 2002-04-25 Ariad Gene Therapeutics, Inc. Methods and materials for regulated production of proteins
AU9036198A (en) 1997-08-26 1999-03-16 Ariad Gene Therapeutics, Inc. Fusion proteins comprising a dimerization, trimerization or tetramerization domain and an additional heterologous transcription activation, transcription repression, dna binding or ligand binding domain
US6479653B1 (en) 1997-08-26 2002-11-12 Ariad Gene Therapeutics, Inc. Compositions and method for regulation of transcription
US6015709A (en) 1997-08-26 2000-01-18 Ariad Pharmaceuticals, Inc. Transcriptional activators, and compositions and uses related thereto
US6333318B1 (en) 1998-05-14 2001-12-25 The Salk Institute For Biological Studies Formulations useful for modulating expression of exogenous genes in mammalian systems, and products related thereto
US6258603B1 (en) 1998-06-17 2001-07-10 Rohm And Haas Company Ligands for modulating the expression of exogenous genes via an ecdysone receptor complex
US20040033600A1 (en) 2001-03-21 2004-02-19 Palli Subba Reddy Ecdysone receptor-based inducible gene expression system
AU2001249315A1 (en) 2000-03-22 2001-10-03 Rheogene, Inc. Ecdysone receptor-based inducible gene expression system
US6436703B1 (en) 2000-03-31 2002-08-20 Hyseq, Inc. Nucleic acids and polypeptides
US8105825B2 (en) 2000-10-03 2012-01-31 Intrexon Corporation Multiple inducible gene regulation system
DK1572862T3 (da) 2001-02-20 2012-11-26 Intrexon Corp Kimære, retinoide x-receptorer og deres anvendelse i et hidtil ukendt ecdysonrecepttorbaseret inducerbart genekspressionssystem
EP1373470B1 (en) 2001-02-20 2013-04-24 Intrexon Corporation Novel substitution mutant receptors and their use in a nuclear receptor-based inducible gene expression system
WO2002066615A2 (en) 2001-02-20 2002-08-29 Rheogene, Inc. Novel substitution mutant receptors and their use in a nuclear receptor-based inducible gene expression system
MXPA03007493A (es) 2001-02-20 2004-10-15 Rheogene Holdings Inc Nuevo sistema de expresion genetica inducible a base de receptor de ecdisona/receptor x retinoide de invertebrado.
US20070060743A1 (en) 2001-05-16 2007-03-15 Tang Y Tom Novel nucleic acids and polypeptides
CA2459807C (en) 2001-09-26 2013-09-03 Jianzhong Zhang Whitefly ecdysone receptor nucleic acids, polypeptides, and uses thereof
MXPA04002810A (es) 2001-09-26 2005-06-06 Rheogene Holdings Inc Acidos nucleicos receptores de ecdisona de saltarilla, polipeptidos, y sus usos.
US20050228016A1 (en) 2002-06-13 2005-10-13 Enrique Michelotti Tetrahydroquinolines for modulating the expression of exogenous genes via an ecdysone receptor complex
US7375093B2 (en) 2002-07-05 2008-05-20 Intrexon Corporation Ketone ligands for modulating the expression of exogenous genes via an ecdysone receptor complex
US20040049437A1 (en) 2002-09-11 2004-03-11 Govone Solutions, Lp Method, system and computer program product for automating transaction tax calculation
US20080050808A1 (en) 2002-10-09 2008-02-28 Reed Thomas D DNA modular cloning vector plasmids and methods for their use
US7785871B2 (en) 2002-10-09 2010-08-31 Intrexon Corporation DNA cloning vector plasmids and methods for their use
US7304161B2 (en) 2003-02-10 2007-12-04 Intrexon Corporation Diaclhydrazine ligands for modulating the expression of exogenous genes in mammalian systems via an ecdysone receptor complex
US7304162B2 (en) 2003-02-21 2007-12-04 Intrexon Corporation Oxadiazoline ligands for modulating the expression of exogenous genes via an ecdysone receptor complex
US7456315B2 (en) 2003-02-28 2008-11-25 Intrexon Corporation Bioavailable diacylhydrazine ligands for modulating the expression of exogenous genes via an ecdysone receptor complex
US7935510B2 (en) 2004-04-30 2011-05-03 Intrexon Corporation Mutant receptors and their use in a nuclear receptor-based inducible gene expression system
EP1751298A4 (en) 2004-05-18 2009-11-11 Intrexon Corp METHOD FOR DYNAMIC VECTOR ASSEMBLY OF DNA CLONING VECTOR PLASMIDES
MX2007007408A (es) 2004-12-20 2007-07-12 Wyeth Corp Analogos de rapamicina y los usos de los mismos en el tratamiento de trastornos neurologicos, proliferativos e inflamatorios.
JP2008524232A (ja) 2004-12-20 2008-07-10 ワイス ラパマイシン誘導体及び神経障害の治療におけるその使用
CA2689137C (en) 2007-05-29 2017-05-02 Intrexon Corporation Chiral diacylhydrazine ligands for modulating the expression of exogenous genes via an ecdysone receptor complex
DK3156414T3 (da) 2007-09-26 2020-03-09 Intrexon Corp Syntetisk 5'utrs ekspressionsvektorer og fremgangsmåder til øgning af transgen ekspression
CN103038343A (zh) 2010-03-23 2013-04-10 英特瑞克斯顿股份有限公司 条件表达治疗蛋白的载体，包含所述载体的宿主细胞，及其应用
DK2826860T3 (en) 2010-04-23 2018-12-03 Univ Massachusetts CNS targeting AAV vectors and methods for their use
EP3012267A1 (en) 2010-07-28 2016-04-27 Roberto Testi Frataxin mutants
WO2012122025A2 (en) * 2011-03-04 2012-09-13 Intrexon Corporation Vectors conditionally expressing protein
EP2500434A1 (en) 2011-03-12 2012-09-19 Association Institut de Myologie Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery
ES2653247T3 (es) 2011-06-09 2018-02-06 Curna, Inc. Tratamiento de enfermedades relacionadas con la frataxina (FXN) mediante inhibición del transcrito antisentido natural al gen FXN
WO2012174452A1 (en) 2011-06-17 2012-12-20 Shire Human Genetic Therapies, Inc. Mitochondrial targeting and therapeutic use thereof
US10640785B2 (en) 2011-11-22 2020-05-05 The Children's Hospital Of Philadelphia Virus vectors for highly efficient transgene delivery
US9066966B2 (en) 2013-02-01 2015-06-30 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods and pharmaceutical compositions for the treatment of cardiomyopathy due to friedreich ataxia
SG11201600987TA (en) * 2013-08-16 2016-03-30 Rana Therapeutics Inc Compositions and methods for modulating rna
WO2015060722A1 (en) 2013-10-24 2015-04-30 Uniqure Ip B.V. Aav-5 pseudotyped vector for gene therapy for neurological diseases
US10975391B2 (en) 2014-04-25 2021-04-13 University Of Massachusetts Recombinant AAV vectors useful for reducing immunity against transgene products
EP3245221A4 (en) 2015-01-16 2018-06-13 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
US20180245073A1 (en) 2015-02-23 2018-08-30 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
WO2016150964A1 (en) 2015-03-23 2016-09-29 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical composition for the treatment and the prevention of neurological phenotype associated with friedreich ataxia
US10617770B2 (en) 2015-04-24 2020-04-14 University Of Florida Research Foundation, Incorporated AAV vector for treatment of Friedreich's ataxia
RU2021102893A (ru) 2015-11-05 2021-03-03 Бамбу Терапьютикс, Инк. Модифицированные гены атаксии фридрейха и векторы для генной терапии
IL300254A (en) 2016-02-05 2023-03-01 Univ Emory Administration of single-stranded or self-complementary adeno-associated virus 9 by injection into cerebrospinal fluid for the purposes of gene therapy in the central nervous system
WO2017165167A1 (en) 2016-03-23 2017-09-28 The Regents Of The University Of California Methods of treating mitochondrial disorders
US20200206361A1 (en) 2016-03-23 2020-07-02 The Regents Of The University Of California Methods of treating mitochondrial disorders
EP3443001A4 (en) 2016-04-11 2020-04-29 Obsidian Therapeutics, Inc. REGULATED BIOCIRCUIT SYSTEMS
CA3029119A1 (en) 2016-06-29 2018-01-04 Crispr Therapeutics Ag Materials and methods for treatment of friedreich ataxia and other related disorders
EP4271420A1 (en) * 2021-01-04 2023-11-08 University Of Florida Research Foundation, Incorporated Methods and compositions for treatment of friedreich's ataxia

Also Published As

Publication number Publication date
US11116852B2 (en) 2021-09-14
US20210177991A1 (en) 2021-06-17
IL266358A (en) 2019-06-30
AU2017359342B2 (en) 2022-02-17
US20180140721A1 (en) 2018-05-24
EP3538660A1 (en) 2019-09-18
SA519401756B1 (ar) 2022-03-20
AU2017359342A1 (en) 2019-05-16
BR112019009446A2 (pt) 2019-07-30
NZ752941A (en) 2023-04-28
EA201990864A1 (ru) 2019-11-29
ZA201902870B (en) 2022-12-21
US20240000973A1 (en) 2024-01-04
CO2019005611A2 (es) 2019-08-20
CN110139933B (zh) 2024-03-08
WO2018089527A1 (en) 2018-05-17
CN110139933A (zh) 2019-08-16
CA3042706A1 (en) 2018-05-17

Similar Documents

Publication Publication Date Title
SA519401756B1 (ar) أجزاء منشأة للتعبير الوراثي عن رنح فريدريك
PH12018500964A1 (en) Modified friedreich ataxia genes and vectors for gene therapy
ZA202107931B (en) Anti-tau antibodies and methods of use
MX2022006260A (es) Compuestos, composiciones y metodos para el tratamiento de enfermedades.
MX2022013526A (es) Degradadores de proteinas y usos de los mismos.
NZ753515A (en) Methods for treatment of cancer comprising tigit-binding agents
MY194032A (en) Anti-tigit antibodies, anti-pvrig antibodies and combinations thereof
PH12019500095A1 (en) Compounds, compositions, and methods for the treatment of disease
PH12017500861A1 (en) Anti-interleukin-33 antibodies and uses thereof
MD3209310T2 (ro) Compoziții care conțin tulpini bacteriene
CR20230163A (es) ANTICUERPOS ANTITAU Y MÉTODOS DE USO (Divisional 2019-0271)
MA39285A (fr) Peptides macrocycliques utiles comme immunomoldulateurs
MX2019006330A (es) Anticuerpos anti-tau y metodos de uso.
MX2018009325A (es) Compuestos y metodos para tratar enfermedades mediadas por el acido ribonucleico (arn).
EP3286321A4 (en) Aav vector for treatment of friedreich's ataxia
MY196448A (en) Anti-Htra1 Antibodies and Methods of use Thereof
MY187366A (en) A method of treating hair using a composition comprising an alpha-substituted aldehyde
MX2020006297A (es) Variantes de cd19.
MX2020003762A (es) Composiciones cosmeticas y metodo para tratar la piel.
SG11202001206YA (en) Emulsions, methods and uses thereof
MX2017010883A (es) Composicion para el tratamiento de la infertilidad.
MY187376A (en) Composition comprising alpha-substituted aldehyde and use thereof with dyed hair
MX2018005987A (es) Compuestos heterociclicos para el tratamiento de enfermedades.
HK1251512A1 (zh) 靶向蛋白造影劑、其製備方法及用途
AU201611534S (en) Hand dryer