ECSP23057004A - Composiciones y métodos para tratar la pérdida de audición y/o la pérdida de visión asociada a clrn1 - Google Patents
Composiciones y métodos para tratar la pérdida de audición y/o la pérdida de visión asociada a clrn1Info
- Publication number
- ECSP23057004A ECSP23057004A ECSENADI202357004A ECDI202357004A ECSP23057004A EC SP23057004 A ECSP23057004 A EC SP23057004A EC SENADI202357004 A ECSENADI202357004 A EC SENADI202357004A EC DI202357004 A ECDI202357004 A EC DI202357004A EC SP23057004 A ECSP23057004 A EC SP23057004A
- Authority
- EC
- Ecuador
- Prior art keywords
- methods
- constructs
- clrn1
- compositions
- loss
- Prior art date
Links
- 206010011878 Deafness Diseases 0.000 title abstract 3
- 208000016354 hearing loss disease Diseases 0.000 title abstract 3
- 238000000034 method Methods 0.000 title abstract 3
- 201000004569 Blindness Diseases 0.000 title abstract 2
- 102100031060 Clarin-1 Human genes 0.000 title abstract 2
- 231100000888 hearing loss Toxicity 0.000 title abstract 2
- 230000010370 hearing loss Effects 0.000 title abstract 2
- 230000004393 visual impairment Effects 0.000 title abstract 2
- 101000992973 Homo sapiens Clarin-1 Proteins 0.000 title 1
- 239000000203 mixture Substances 0.000 title 1
- 108091026890 Coding region Proteins 0.000 abstract 2
- 101710093463 Clarin-1 Proteins 0.000 abstract 1
- 231100000895 deafness Toxicity 0.000 abstract 1
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
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/16—Otologicals
-
- 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
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
-
- 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
- A61K48/0091—Purification or manufacturing processes for gene therapy compositions
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
-
- 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
-
- 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
- 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
-
- 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
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
-
- 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
- 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
- C12N2750/14151—Methods of production or purification of viral material
- C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
-
- 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
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
-
- 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
- C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
-
- 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
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
-
- 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
- C12N2840/44—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor
- C12N2840/445—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor for trans-splicing, e.g. polypyrimidine tract, branch point splicing
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biochemistry (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biophysics (AREA)
- Epidemiology (AREA)
- General Chemical & Material Sciences (AREA)
- Microbiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Cell Biology (AREA)
- Plant Pathology (AREA)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- Ophthalmology & Optometry (AREA)
- Manufacturing & Machinery (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Abstract
La presente descripción proporciona construcciones que comprenden una secuencia codificante unida de forma operativa a un promotor, donde la secuencia codificante codifica una proteína clarin 1. Las construcciones de ejemplo incluyen construcciones de AAV. También se proporcionan métodos para usar construcciones descritas para el tratamiento de la pérdida de audición y/o sordera. También se proporcionan métodos para usar construcciones descritas para el tratamiento de la pérdida de visión.
Applications Claiming Priority (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US202063131413P | 2020-12-29 | 2020-12-29 |
Publications (1)
Publication Number | Publication Date |
---|---|
ECSP23057004A true ECSP23057004A (es) | 2023-08-31 |
Family
ID=80050943
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
ECSENADI202357004A ECSP23057004A (es) | 2020-12-29 | 2023-07-27 | Composiciones y métodos para tratar la pérdida de audición y/o la pérdida de visión asociada a clrn1 |
Country Status (15)
Country | Link |
---|---|
US (1) | US20240139344A1 (es) |
EP (1) | EP4271419A1 (es) |
JP (1) | JP2024500786A (es) |
KR (1) | KR20230127263A (es) |
CN (1) | CN117157108A (es) |
AU (1) | AU2021412962A1 (es) |
CA (1) | CA3203373A1 (es) |
CL (1) | CL2023001896A1 (es) |
CO (1) | CO2023007325A2 (es) |
DO (1) | DOP2023000132A (es) |
EC (1) | ECSP23057004A (es) |
IL (1) | IL304006A (es) |
MX (1) | MX2023007800A (es) |
PE (1) | PE20240012A1 (es) |
WO (1) | WO2022146839A1 (es) |
Family Cites Families (37)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
EP0173552B1 (en) | 1984-08-24 | 1991-10-09 | The Upjohn Company | Recombinant dna compounds and the expression of polypeptides such as tpa |
US5168062A (en) | 1985-01-30 | 1992-12-01 | University Of Iowa Research Foundation | Transfer vectors and microorganisms containing human cytomegalovirus immediate-early promoter-regulatory DNA sequence |
US4797368A (en) | 1985-03-15 | 1989-01-10 | The United States Of America As Represented By The Department Of Health And Human Services | Adeno-associated virus as eukaryotic expression vector |
US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
US5436146A (en) | 1989-09-07 | 1995-07-25 | The Trustees Of Princeton University | Helper-free stocks of recombinant adeno-associated virus vectors |
US6268213B1 (en) | 1992-06-03 | 2001-07-31 | Richard Jude Samulski | Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy |
US5869305A (en) | 1992-12-04 | 1999-02-09 | The University Of Pittsburgh | Recombinant viral vector system |
US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
US5741683A (en) | 1995-06-07 | 1998-04-21 | The Research Foundation Of State University Of New York | In vitro packaging of adeno-associated virus DNA |
US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
US6001650A (en) | 1995-08-03 | 1999-12-14 | Avigen, Inc. | High-efficiency wild-type-free AAV helper functions |
WO1998010088A1 (en) | 1996-09-06 | 1998-03-12 | Trustees Of The University Of Pennsylvania | An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase |
JP4135120B2 (ja) | 1997-04-14 | 2008-08-20 | セル ジェネシス,インコーポレーテッド | 組換えaav生産の効率を上昇させる方法 |
US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
EP1080218A1 (en) | 1998-05-27 | 2001-03-07 | University of Florida | Method of preparing recombinant adeno-associated virus compositions by using an iodixanol gradient |
WO2000028004A1 (en) | 1998-11-10 | 2000-05-18 | The University Of North Carolina At Chapel Hill | Virus vectors and methods of making and administering the same |
JP2004501113A (ja) | 2000-06-01 | 2004-01-15 | ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル | 組み換えパルボウィルスベクターの制御放出のための方法および配合物 |
ES2345876T3 (es) | 2001-05-31 | 2010-10-05 | Novartis Vaccines And Diagnostics, Inc. | Particulas de replicones de alfavirus quimericos. |
US7045335B2 (en) | 2001-09-06 | 2006-05-16 | Alphavax, Inc. | Alphavirus replicon vector systems |
NZ600121A (en) | 2001-11-13 | 2013-12-20 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
AU2002360291A1 (en) | 2001-12-17 | 2003-06-30 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) serotype 8 sequences |
WO2003104413A2 (en) | 2002-06-05 | 2003-12-18 | University Of Florida | Production of pseudotyped recombinant aav virions |
JP5054975B2 (ja) | 2003-09-30 | 2012-10-24 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | アデノ随伴ウイルス(aav)の同源系統群、配列、それらを含有するベクターおよびそれらの用途 |
US7557197B2 (en) | 2004-01-28 | 2009-07-07 | Oregon Health & Science University | Human soluble neuropilin-1 primary polyadenylation signal and uses thereof |
US20060040354A1 (en) | 2004-07-20 | 2006-02-23 | O'keefe Theresa L | Novel polyadenylation signal for use in expression vectors |
JP5702519B2 (ja) | 2005-04-07 | 2015-04-15 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | Aavベクターの機能を上げる方法 |
WO2007120542A2 (en) | 2006-03-30 | 2007-10-25 | The Board Of Trustees Of The Leland Stanford Junior University | Aav capsid library and aav capsid proteins |
WO2013148302A1 (en) | 2012-03-26 | 2013-10-03 | THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES, OFFICE OF TECHNOLOGY TRANSFER, NATIONAL INSTITUTES OF HEALTH | Delivery of packaged rna to mammalian cells |
GB201518979D0 (en) | 2015-10-27 | 2015-12-09 | Univ Leuven Kath | Treatment of hepatic steatosis related oligo-ovulation |
US10201455B2 (en) | 2016-06-22 | 2019-02-12 | The Charles Stark Draper Laboratory, Inc. | System for inner ear drug delivery via trans-round window membrane injection |
CA3034527A1 (en) | 2016-08-23 | 2018-03-01 | Emmanuel John Simons | Compositions and methods for treating non-age-associated hearing impairment in a human subject |
US11439749B2 (en) | 2017-10-25 | 2022-09-13 | The Charles Stark Draper Laboratory, Inc. | Apparatus and method for trans-round window membrane drug delivery |
EA202091995A1 (ru) | 2018-02-22 | 2021-03-30 | Акуос, Инк. | Композиции и способы лечения не связанного с возрастом нарушения слуха у субъекта-человека |
US20210079406A1 (en) * | 2018-04-10 | 2021-03-18 | President And Fellows Of Harvard College | Aav vectors encoding clarin-1 or gjb2 and uses thereof |
CN113853437A (zh) | 2018-11-07 | 2021-12-28 | 阿库斯股份有限公司 | 腺相关病毒载体用于在内耳中的毛细胞和支持细胞中校正基因缺陷/表达蛋白质的用途 |
BR112022023974A2 (pt) | 2020-05-27 | 2022-12-20 | Akouos Inc | Dispositivos, sistemas e métodos para entregar fluido para o ouvido interno |
-
2021
- 2021-12-22 CA CA3203373A patent/CA3203373A1/en active Pending
- 2021-12-22 AU AU2021412962A patent/AU2021412962A1/en active Pending
- 2021-12-22 KR KR1020237025236A patent/KR20230127263A/ko unknown
- 2021-12-22 EP EP21848394.9A patent/EP4271419A1/en active Pending
- 2021-12-22 WO PCT/US2021/064924 patent/WO2022146839A1/en active Application Filing
- 2021-12-22 MX MX2023007800A patent/MX2023007800A/es unknown
- 2021-12-22 US US18/269,307 patent/US20240139344A1/en active Pending
- 2021-12-22 JP JP2023537130A patent/JP2024500786A/ja active Pending
- 2021-12-22 CN CN202180088219.XA patent/CN117157108A/zh active Pending
- 2021-12-22 IL IL304006A patent/IL304006A/en unknown
- 2021-12-22 PE PE2023001959A patent/PE20240012A1/es unknown
-
2023
- 2023-06-02 CO CONC2023/0007325A patent/CO2023007325A2/es unknown
- 2023-06-23 CL CL2023001896A patent/CL2023001896A1/es unknown
- 2023-06-26 DO DO2023000132A patent/DOP2023000132A/es unknown
- 2023-07-27 EC ECSENADI202357004A patent/ECSP23057004A/es unknown
Also Published As
Publication number | Publication date |
---|---|
KR20230127263A (ko) | 2023-08-31 |
EP4271419A1 (en) | 2023-11-08 |
JP2024500786A (ja) | 2024-01-10 |
CN117157108A (zh) | 2023-12-01 |
PE20240012A1 (es) | 2024-01-04 |
DOP2023000132A (es) | 2023-07-31 |
MX2023007800A (es) | 2023-07-11 |
CA3203373A1 (en) | 2022-07-07 |
IL304006A (en) | 2023-08-01 |
CO2023007325A2 (es) | 2023-06-20 |
US20240139344A1 (en) | 2024-05-02 |
WO2022146839A1 (en) | 2022-07-07 |
AU2021412962A1 (en) | 2023-08-17 |
CL2023001896A1 (es) | 2023-12-15 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
CL2022003147A1 (es) | Composiciones y métodos para tratar pérdida auditiva asociada con gjb2 | |
CL2022001375A1 (es) | Receptores de antígeno quimérico cd19 y cd22 y usos de los mismos | |
MX2020003557A (es) | Terapias genicas para los trastornos lisosomales. | |
MX2021008131A (es) | Metodos y composiciones para el tratamiento de la enfermedad de fabry. | |
EA202091995A1 (ru) | Композиции и способы лечения не связанного с возрастом нарушения слуха у субъекта-человека | |
CL2019002368A1 (es) | Composiciones y métodos para el tratamiento de cáncer. | |
CO2020016718A2 (es) | Terapia génica cardíaca con aav para cardiomiopatía | |
CL2019001749A1 (es) | Uso de anticuerpos anti-esclerostina en el tratamiento de osteogenesis imperfecta. | |
AR121390A1 (es) | Composiciones y métodos para tratar la deficiencia auditiva no relacionada con la edad en un sujeto humano | |
CO2020013690A2 (es) | Composiciones y métodos para el tratamiento de la distrofia macular | |
ECSP20055797A (es) | Métodos y composiciones para tratar enfermedades pulmonares crónicas | |
CL2022003153A1 (es) | Composiciones y métodos para tratar la pérdida auditiva asociada a slc26a4 | |
MX2019007788A (es) | Composiciones y metodos para tratar condiciones de la piel mediante el uso de luz y clorhidrato de glucosamina. | |
ECSP23057004A (es) | Composiciones y métodos para tratar la pérdida de audición y/o la pérdida de visión asociada a clrn1 | |
CO2021003136A2 (es) | Terapias de combinación | |
CO2022008738A2 (es) | Célula vectorial adyuvante artificial que contiene nyeso- 1 para uso en el tratamiento de cáncer | |
BR112023020490A2 (pt) | Composições e métodos para expressão de transgenes oculares | |
ECSP23093588A (es) | Composiciones de suministro de terapia génica y métodos para tratar perdida auditiva | |
CO2021005872A2 (es) | Composiciones y métodos para la estimulación ovárica controlada | |
DOP2020000049A (es) | MÉTODOS PARA EL TRATAMIENTO DE ENFERMEDADES RELACIONADAS CON TNFa | |
CO2020008885A2 (es) | Arilsulfatasa a purificada y composiciones de la misma | |
CL2022002406A1 (es) | Composiciones sinérgicas y dirigidas para el tratamiento de trombosis arterial y venosa | |
AR120198A1 (es) | Construcciones de variantes de igf2 | |
CO2022003324A2 (es) | Anticuerpos anti-nrp1a y sus usos para tratar enfermedades oculares | |
ECSP22020678A (es) | Anticuerpos anti-nrp1a y sus usos para tratar enfermedades oculares |