CO2021006957A2 - Método para el tratamiento de la distrofia muscular mediante el gen de utrofina - Google Patents

Método para el tratamiento de la distrofia muscular mediante el gen de utrofina

Info

Publication number
CO2021006957A2
CO2021006957A2 CONC2021/0006957A CO2021006957A CO2021006957A2 CO 2021006957 A2 CO2021006957 A2 CO 2021006957A2 CO 2021006957 A CO2021006957 A CO 2021006957A CO 2021006957 A2 CO2021006957 A2 CO 2021006957A2
Authority
CO
Colombia
Prior art keywords
muscular dystrophy
treatment
region
utrophin gene
sequence encoding
Prior art date
Application number
CONC2021/0006957A
Other languages
English (en)
Inventor
Eiji Yoshimi
Katsuro Yoshioka
Tetsuya Yamagata
Yuanbo Qin
Iain Robert Thompson
Nidhi Khanna
Original Assignee
Modalis Therapeutics Corp
Astellas Pharma Inc
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 Modalis Therapeutics Corp, Astellas Pharma Inc filed Critical Modalis Therapeutics Corp
Publication of CO2021006957A2 publication Critical patent/CO2021006957A2/es

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702Regulators; Modulating activity
    • C07K14/4705Regulators; Modulating activity stimulating, promoting or activating activity
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707Muscular dystrophy
    • C07K14/4708Duchenne dystrophy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases RNAses, DNAses
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/16011Human Immunodeficiency Virus, HIV
    • C12N2740/16041Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • C12N2800/80Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

Landscapes

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

Abstract

RESUMEN Polinucleótidos que comprenden las siguientes secuencias de bases: (a) una secuencia de bases que codifica una proteína de fusión de una proteína efectora CRISPR deficiente en nucleasas y un activador de la transcripción, y (b) una secuencia de bases que codifica un ARN guía dirigido a una región continua de 18 a 24 nucleótidos de longitud en una región establecida en SEQ ID NO: 104, 105, 135, 141, 153, 167 o 172 en la región reguladora de la expresión del gen de la utrofina humana Se espera que sean útiles para tratar o prevenir la distrofia muscular de DUCHENNE o la distrofia muscular de BECKER.
CONC2021/0006957A 2018-11-16 2021-05-26 Método para el tratamiento de la distrofia muscular mediante el gen de utrofina CO2021006957A2 (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201862768474P 2018-11-16 2018-11-16
US201962861039P 2019-06-13 2019-06-13
US201962931925P 2019-11-07 2019-11-07
PCT/JP2019/045716 WO2020101042A1 (en) 2018-11-16 2019-11-15 Method for treating muscular dystrophy by targeting utrophin gene

Publications (1)

Publication Number Publication Date
CO2021006957A2 true CO2021006957A2 (es) 2021-06-10

Family

ID=68988228

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2021/0006957A CO2021006957A2 (es) 2018-11-16 2021-05-26 Método para el tratamiento de la distrofia muscular mediante el gen de utrofina

Country Status (15)

Country Link
US (1) US11473071B2 (es)
EP (1) EP3880255A1 (es)
JP (2) JP7069426B2 (es)
KR (1) KR20210093954A (es)
CN (1) CN113271982A (es)
AU (1) AU2019381460A1 (es)
BR (1) BR112021009481A2 (es)
CA (1) CA3119618A1 (es)
CO (1) CO2021006957A2 (es)
IL (1) IL283123A (es)
MX (1) MX2021005720A (es)
PH (1) PH12021551122A1 (es)
SG (1) SG11202105030VA (es)
TW (1) TW202033224A (es)
WO (1) WO2020101042A1 (es)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102008496B1 (ko) * 2013-03-25 2019-08-07 삼성전자주식회사 단일 센서를 이용하여 기구물의 이동 방향 및 속도를 검출하는 방법 및 장치.
EP3781705A4 (en) 2018-04-19 2022-01-26 The Regents of the University of California COMPOSITIONS AND METHODS FOR GENE EDITING
AU2019281158A1 (en) * 2018-06-08 2021-01-14 Modalis Therapeutics Corporation Modified Cas9 protein and use thereof
US20210332094A1 (en) * 2018-08-07 2021-10-28 Modalis Therapeutics Corporation Novel transcription activator
WO2021230385A1 (en) 2020-05-15 2021-11-18 Astellas Pharma Inc. Method for treating muscular dystrophy by targeting utrophin gene
JP2023542728A (ja) 2020-09-29 2023-10-11 ジェネトン ユートロフィン調節エレメント内に変異を誘導することによる細胞におけるユートロフィン発現の増強、及びその治療的使用
KR102421411B1 (ko) 2021-10-29 2022-07-18 주식회사 케이에스비튜젠 옥시라세탐을 포함하는 근이영양증 예방 또는 치료용 조성물
EP4331579A1 (en) 2021-04-26 2024-03-06 KSB Tugen Inc. Composition comprising oxiracetam for preventing or treating muscular disease
CN113563428B (zh) * 2021-06-22 2022-08-02 呈诺再生医学科技(珠海横琴新区)有限公司 特定空间结构多肽及其在制备iPSC中的应用
WO2023190935A1 (en) * 2022-03-31 2023-10-05 Modalis Therapeutics Corporation Method for treating myopathies by targeting titin gene
CN116218849B (zh) * 2023-01-18 2024-01-23 昆明理工大学 针对utrophin激活治疗杜氏肌营养不良的sgRNA和应用

Family Cites Families (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN100422320C (zh) * 2000-04-28 2008-10-01 肖啸 编码抗肌萎缩蛋白小基因的dna序列及其使用方法
EP1390490B1 (en) 2001-05-24 2009-04-15 Genzyme Corporation Muscle-specific expression vectors
EP2338900B1 (en) 2001-11-13 2014-01-01 The Trustees of The University of Pennsylvania Adeno-associated virus (AAV) Cy.5 sequences, vectors containing the same and uses thereof
DK2292779T3 (en) 2003-09-30 2017-02-27 Univ Pennsylvania ADENOASS-ASSOCIATED VIRUS (AAV) CLADES, SEQUENCES, VECTORS CONTAINING SAME AND APPLICATIONS THEREOF
CA2553676A1 (en) 2004-01-28 2005-08-11 Research Development Foundation Human soluble neuropilin-1 primary polyadenylation signal and uses thereof
ITRM20070523A1 (it) 2007-10-05 2009-04-06 Consiglio Nazionale Ricerche Acido nucleico codificante per una proteina regolatrice specifica della trascrizione dell'utrofina proteina da esso codificata e sue applicazioni
US10738326B2 (en) 2010-10-27 2020-08-11 Jichi Medical University Adeno-associated virus vector for gene transfer to nervous system cells
EP2850185A4 (en) * 2012-05-16 2015-12-30 Rana Therapeutics Inc COMPOSITIONS AND METHODS FOR MODULATING UTRN EXPRESSION
UA118014C2 (uk) 2012-05-25 2018-11-12 Те Ріджентс Оф Те Юніверсіті Оф Каліфорнія Спосіб модифікації днк-мішені
KR20230136697A (ko) * 2013-06-05 2023-09-26 듀크 유니버시티 Rna-가이드 유전자 편집 및 유전자 조절
ITTO20130669A1 (it) 2013-08-05 2015-02-06 Consiglio Nazionale Ricerche Vettore adeno-associato ricombinante muscolo-specifico e suo impiego nel trattamento di patologie muscolari
EP3275448A4 (en) 2015-03-24 2019-05-01 Kyowa Hakko Kirin Co., Ltd. LIPID NANOPARTICLES CONTAINING NUCLEIC ACIDS
CA2996982A1 (en) * 2015-09-23 2017-03-30 Universite Laval Modification of the dystrophin gene and uses thereof
CA3048963A1 (en) * 2016-02-11 2017-08-17 The Regents Of The University Of California Methods and compositions for modifying a mutant dystrophin gene in a cell's genome
US11674128B2 (en) * 2016-12-12 2023-06-13 Tsinghua University Engineering of a minimal SaCas9 CRISPR/Cas system for gene editing and transcriptional regulation optimized by enhanced guide RNA
CN110612113B (zh) * 2017-02-07 2024-03-26 加利福尼亚大学董事会 单倍体机能不全的基因疗法
US11674138B2 (en) * 2017-03-13 2023-06-13 President And Fellows Of Harvard College Methods of modulating expression of target nucleic acid sequences in a cell
US20210254056A1 (en) * 2017-05-05 2021-08-19 Camp4 Therapeutics Corporation Identification and targeted modulation of gene signaling networks
US20210363193A1 (en) 2018-04-27 2021-11-25 Universität Heidelberg Modified aav capsid polypeptides for treatment of muscular diseases
WO2019236081A1 (en) * 2018-06-06 2019-12-12 Salk Institute For Biological Studies Targeted gene activation using modified guide rna

Also Published As

Publication number Publication date
SG11202105030VA (en) 2021-06-29
US20210355464A1 (en) 2021-11-18
AU2019381460A1 (en) 2021-06-17
WO2020101042A8 (en) 2020-07-30
IL283123A (en) 2021-06-30
KR20210093954A (ko) 2021-07-28
JP2022115976A (ja) 2022-08-09
BR112021009481A2 (pt) 2021-08-17
JP7069426B2 (ja) 2022-05-17
PH12021551122A1 (en) 2021-11-22
CA3119618A1 (en) 2020-05-22
MX2021005720A (es) 2021-07-21
US11473071B2 (en) 2022-10-18
EP3880255A1 (en) 2021-09-22
CN113271982A (zh) 2021-08-17
JP2022509560A (ja) 2022-01-20
WO2020101042A1 (en) 2020-05-22
TW202033224A (zh) 2020-09-16

Similar Documents

Publication Publication Date Title
CO2021006957A2 (es) Método para el tratamiento de la distrofia muscular mediante el gen de utrofina
CO2021014746A2 (es) Método para tratar distrofia muscular por direccionamiento del gen dmpk
CL2019000732A1 (es) Tratamiento avv de la enfermedad de huntington.
CY1124742T1 (el) 4'-φθορο-νουκλεοσιδια, 4'-φθορο-νουκλοεοτιδια και αναλογα αυτων για τη θεραπευτικη αγωγη toy hcv
WO2016106401A3 (en) Rna agents for p21 gene modulation
MX2017014821A (es) Método para determinar la secuencia de un acido nucleico usando luminiscencia resuelta por tiempo.
CY1124749T1 (el) Παραγωγα διπυραζολιου χρησιμα για την θεραπεια αυτοανοσων νοσηματων
EA201791417A1 (ru) Супрессия гена гентингтина, индуцированная рнк-интерференцией
CO2017012454A2 (es) Secuencias de uricasa adecuadas para el tratamiento de hiperuricemia
EA201892366A1 (ru) Антисмысловые олигомеры и способы их применения для лечения заболеваний, связанных с геном кислой альфа-глюкозидазы
BR112021024463A2 (pt) Oligonucleotídeos e métodos de uso para o tratamento de doenças neurológicas
EA202091995A1 (ru) Композиции и способы лечения не связанного с возрастом нарушения слуха у субъекта-человека
EA202091673A1 (ru) Способ амплификации нуклеиновых кислот
MX2018014163A (es) Composiciones de nucleotido marcados y metodos para el secuenciamiento de acido nucleico.
NZ752941A (en) Frataxin expression constructs
WO2020036654A3 (en) Highly knotted molecular topologies from single-stranded nucleic acids
CO2021000914A2 (es) Productos y métodos de virus adenoasociado recombinante para el tratamiento de la distrofia muscular de cinturas 2a
EA201890379A1 (ru) Терапевтические олигонуклеотиды
EA201791471A1 (ru) Способы применения антисмысловых олигонуклеотидов smad7
PE20211238A1 (es) Tratamiento antisentido del sindrome de angelman
PE20212248A1 (es) Molecula oligomerica de acido nucleico y uso de esta
EA201891134A1 (ru) Способы лечения мышечной дистрофии
BR112022002889A2 (pt) Método para tratar distrofia muscular tendo como alvo o gene lama1
MX2020012278A (es) Ensamblaje de polimero biologico habilitado para aprendizaje de maquina.
BR112016015875A2 (pt) Elementos intensificadores de cpmv