Animal model for genetic diseases

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A01K2267/0306

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AHUMAN NECESSITIES A01Agriculture A01KANIMAL HUSBANDRY CARE OF BIRDS, FISHES, INSECTS FISHING REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR NEW BREEDS OF ANIMALS A01K2267/00Animals characterised by purpose

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A01K2267/0306Animal model for genetic diseases

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Patents Grantslast 30 patents

Information Patent Grant
Transgenic animal having CREBBP gene into which mutation has been i...
- Patent number 12,108,746
- Issue date Oct 8, 2024
- Kurume University
- Takahiro Sato
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Non-human animals models of retinoschisis
- Patent number 12,082,565
- Issue date Sep 10, 2024
- REGENERON PHARMACEUTICALS, INC.
- Susannah Brydges
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
Information Patent Grant
Treatment for autistic spectrum disorder (ASD) and relevant symptoms
- Patent number 12,059,433
- Issue date Aug 13, 2024
- Academia Sinica
- Yi-Ping Hsueh
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Antisense compounds targeting genes associated with cystic fibrosis
- Patent number 12,042,508
- Issue date Jul 23, 2024
- Rosalind Franklin University of Medicine and Science
- Michelle L. Hastings
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
Information Patent Grant
Cell therapy with lentiviral transduced CSF2RA transgene in the tre...
- Patent number 12,043,844
- Issue date Jul 23, 2024
- CHILDREN'S HOSPITAL MEDICAL CENTER
- Bruce C. Trapnell
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
Information Patent Grant
Non-human animals comprising a humanized TTR locus and methods of use
- Patent number 12,010,979
- Issue date Jun 18, 2024
- REGENERON PHARMACEUTICALS, INC.
- Meghan Drummond Samuelson
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
Information Patent Grant
Drug for retinal degenerative disease associated with photoreceptor...
- Patent number 11,931,327
- Issue date Mar 19, 2024
- Daiichi Sankyo Company, Limited
- Naoki Tsuji
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
Information Patent Grant
Rat model of down syndrome and method for producing same
- Patent number 11,825,817
- Issue date Nov 28, 2023
- National University Corporation Tottori University
- Yasuhiro Kazuki
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Methods of treating muscular dystrophy
- Patent number 11,820,972
- Issue date Nov 21, 2023
- Research Institute at Nationwide Children's Hospital
- Louise Rodino-Klapac
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Mutations in rhodopsin gene in zebrafish and uses thereof
- Patent number 11,746,391
- Issue date Sep 5, 2023
- The Florida State University Research Foundation, Inc.
- James Fadool
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
Information Patent Grant
Methods and compositions for treating chronic granulomatous disease
- Patent number 11,738,053
- Issue date Aug 29, 2023
- Board of Regents, The University of Texas System
- Senlin Li
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Fibrillin-1 mutations for modeling neonatal progeroid syndrome with...
- Patent number 11,730,150
- Issue date Aug 22, 2023
- REGENERON PHARMACEUTICALS, INC.
- Charleen Hunt
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
Information Patent Grant
Prevention and/or treatment of hearing loss or impairment
- Patent number 11,679,140
- Issue date Jun 20, 2023
- Institut Pasteur
- Christine Petit
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Methods and compositions for modifying a mutant dystrophin gene in...
- Patent number 11,666,666
- Issue date Jun 6, 2023
- The Regents of the University of California
- April D. Pyle
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Method of making a rat model of retinal degeneration and rat model...
- Patent number 11,653,636
- Issue date May 23, 2023
- University of Ulsan Foundation for Industry Cooperation
- Joo Yong Lee
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Methods and compositions for modified factor IX fusion proteins
- Patent number 11,560,418
- Issue date Jan 24, 2023
- The University of North Carolina at Chapel Hill
- Tal Kafri
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Optimized mini-dystrophin genes and expression cassettes and their use
- Patent number 11,547,765
- Issue date Jan 10, 2023
- The University of North Carolina at Chapel Hill
- Xiao Xiao
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
Information Patent Grant
Transcription modulation in animals using CRISPR/Cas systems
- Patent number 11,519,004
- Issue date Dec 6, 2022
- Regeneran Pharmaceuticals, Inc.
- Charleen Hunt
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
Information Patent Grant
Use and production of CHD8+/− transgenic animals with behavioral ph...
- Patent number 11,459,557
- Issue date Oct 4, 2022
- The Broad Institute, Inc.
- Randall Jeffrey Platt
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
Information Patent Grant
Anti-factor D antibodies and uses thereof
- Patent number 11,434,279
- Issue date Sep 6, 2022
- The Trustees of the University of Pennsylvania
- Wenchao Song
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Compositions and methods for enhanced genome editing
- Patent number 11,427,837
- Issue date Aug 30, 2022
- The Regents of the University of California
- Jacob Corn
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Genetically modified mouse with an inducible ACVR1 gene with a muta...
- Patent number RE49121
- Issue date Jul 5, 2022
- REGENERON PHARMACEUTICALS, INC.
- Aris N. Economides
Information Patent Grant
Methods of reducing FUS/TLS- or TDP-43-mediated neuronal cytotoxici...
- Patent number 11,332,504
- Issue date May 17, 2022
- Brandeis University
- Greg Petsko
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Large animal model for developing therapeutic agents to treat impai...
- Patent number 11,278,013
- Issue date Mar 22, 2022
- Usher 2020 Foundation
- Uwe Wolfrum
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Targeted RNA knockdown and knockout by type III-A Csm complexes
- Patent number 11,274,317
- Issue date Mar 15, 2022
- Vilnius University
- Thomas Fricke
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
Information Patent Grant
Disease model pig exhibiting stable phenotype, and production metho...
- Patent number 11,246,299
- Issue date Feb 15, 2022
- PORMEDTEC CO., LTD.
- Hiroshi Nagashima
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
Information Patent Grant
Delivery, use and therapeutic applications of the CRISPR-Cas system...
- Patent number 11,124,796
- Issue date Sep 21, 2021
- The Broad Institute, Inc.
- Phillip A. Sharp
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
Information Patent Grant
Antisense compounds targeting genes associated with cystic fibrosis
- Patent number 11,116,785
- Issue date Sep 14, 2021
- Rosalind Franklin University of Medicine and Science
- Michelle L. Hastings
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Methods for the treatment of Danon disease and other disorders of a...
- Patent number 11,065,347
- Issue date Jul 20, 2021
- The Regents of the University of California
- Eric D. Adler
- C07 - ORGANIC CHEMISTRY
Information Patent Grant
Non-human animal models of retinoschisis
- Patent number 11,064,685
- Issue date Jul 20, 2021
- REGENERON PHARMACEUTICALS, INC.
- Susannah Brydges
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING

Patents Applicationslast 30 patents

Information Patent Application
HUMANIZED VIPR2 COPY NUMBER VARIANT TRANSGENIC MOUSE MODEL FOR ANTI...
- Publication number 20240392312
- Publication date Nov 28, 2024
- Board of Supervisors of Louisiana State University and Agricultural and Mecha...
- Xiaohong LIU
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
Information Patent Application
IMMUNOTHERAPY COMPOSITIONS AND METHODS FOR TREATMENT OF TAUOPATHY A...
- Publication number 20240299512
- Publication date Sep 12, 2024
- UNM Rainforest Innovations
- Kiran Bhaskar
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS OF TREATMENT WITH AMINOLEVULINIC ACID SYNTHASE 2 (ALAS2) MO...
- Publication number 20240276957
- Publication date Aug 22, 2024
- AGIOS PHARMACEUTICALS, INC
- Giovanni Cianchetta
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
Information Patent Application
Use Of Citicoline In Regulating Respiratory Chain Function Related...
- Publication number 20240269160
- Publication date Aug 15, 2024
- Tongji Hospital, Tongji Medical College, Huazhong University of Science and T...
- Chen CHEN
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
GENE THERAPY CONSTRUCTS AND METHODS FOR TREATMENT OF HEARING LOSS
- Publication number 20240252681
- Publication date Aug 1, 2024
- Rescue Hearing Inc
- Hinrich Staecker
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
MOUSE MODEL AND TREATMENT OF HEREDITARY INCLUSION BODY MYOPATHY
- Publication number 20240216405
- Publication date Jul 4, 2024
- HIBM RESEARCH GROUP, INC.
- Daniel Kohan DARVISH
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
Information Patent Application
IMMUNOTHERAPY COMPOSITIONS AND METHODS FOR TREATMENT OF TAUOPATHY A...
- Publication number 20240197845
- Publication date Jun 20, 2024
- UNM Rainforest Innovations
- Kiran Bhaskar
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
Information Patent Application
Methods of Treating Muscular Dystrophy
- Publication number 20240191250
- Publication date Jun 13, 2024
- Research Institute at Nationwide Children's Hospital
- Louise Rodino-Klapac
- C07 - ORGANIC CHEMISTRY
Information Patent Application
Methods and Compositions for Modifying a Mutant Dystrophin Gene in...
- Publication number 20240156989
- Publication date May 16, 2024
- The Regents of the University of California
- April D. Pyle
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
DRUG FOR RETINAL DEGENERATIVE DISEASE ASSOCIATED WITH PHOTORECEPTOR...
- Publication number 20240139134
- Publication date May 2, 2024
- DAIICHI SANKYO COMPANY, LIMITED
- Naoki Tsuji
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
PREVENTION AND/OR TREATMENT OF HEARING LOSS OR IMPAIRMENT
- Publication number 20230405085
- Publication date Dec 21, 2023
- INSTITUT PASTEUR
- Christine PETIT
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
OPTIMIZED MINI-DYSTROPHIN GENES AND EXPRESSION CASSETTES AND THEIR USE
- Publication number 20230398236
- Publication date Dec 14, 2023
- The University of North Carolina at Chapel Hill
- Xiao Xiao
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
A HIGH-THROUGHPUT SCREENING METHOD TO DISCOVER OPTIMAL GRNA PAIRS F...
- Publication number 20230349888
- Publication date Nov 2, 2023
- DUKE UNIVERSITY
- Charles A. Gersbach
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
Information Patent Application
NOVEL METHOD FOR PREPARING ANIMAL MODEL OF CEREBROVASCULAR DISEASE...
- Publication number 20230309521
- Publication date Oct 5, 2023
- Dongguk University Industry-Academic Cooperation Foundation
- Dong Eog KIM
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
Information Patent Application
GENE THERAPY FOR NIEMANN-PICK DISEASE TYPE A
- Publication number 20230287366
- Publication date Sep 14, 2023
- Genzyme Corporation
- Marco A. PASSINI
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
GENETICALLY MODIFIED NON-HUMAN ANIMAL WITH HUMAN OR CHIMERIC CD38
- Publication number 20230148574
- Publication date May 18, 2023
- Biocytogen Pharmaceuticals (Beijing) Co., Ltd.
- Yuelei Shen
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
TRANSCRIPTION MODULATION IN ANIMALS USING CRISPR/CAS SYSTEMS
- Publication number 20230123296
- Publication date Apr 20, 2023
- Regeneron Pharmaceuticals, Inc.
- Charleen Hunt
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
Information Patent Application
METHODS OF CONTROLLING BODYWEIGHT BY MODULATING PHOSPHATIDYLINOSITO...
- Publication number 20230096781
- Publication date Mar 30, 2023
- University of Cincinnati
- Atsuo Sasaki
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
NON-HUMAN ANIMALS COMPRISING A HUMANIZED TTR LOCUS AND METHODS OF USE
- Publication number 20230078551
- Publication date Mar 16, 2023
- Regeneron Pharmaceuticals, Inc.
- Meghan Drummond-Samuelson
- C07 - ORGANIC CHEMISTRY
Information Patent Application
TREATMENT OF MUCOPOLYSACCHARIDOSIS IVA
- Publication number 20230064077
- Publication date Mar 2, 2023
- Regenxbio Inc.
- Devin MCDOUGALD
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
Information Patent Application
TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS
- Publication number 20230054101
- Publication date Feb 23, 2023
- Brandeis University
- Greg Petsko
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
Information Patent Application
ANTI-FACTOR D ANTIBODIES AND USES THEREOF
- Publication number 20230032737
- Publication date Feb 2, 2023
- The Trustees of the University of Pennsylvania
- Wenchao Song
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
COMPOSITIONS AND METHODS FOR GENE REPLACEMENT
- Publication number 20220395583
- Publication date Dec 15, 2022
- Children's Medical Center Corporation
- Jeffrey R. HOLT
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
TRANSGENIC NON-HUMAN ANIMAL CAPABLE OF CONTROLLING EXPRESSION OF TR...
- Publication number 20220354970
- Publication date Nov 10, 2022
- TOKYO METROPOLITAN INSTITUTE OF MEDICAL SCIENCE
- Haruo OKADO
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
Information Patent Application
Genetically Modified Non-Human Animals and Methods of Use Thereof
- Publication number 20220354098
- Publication date Nov 10, 2022
- Yale University
- Richard Flavell
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
Information Patent Application
COMPOSITIONS AND METHODS FOR TREATING SENSORINEURAL HEARING LOSS US...
- Publication number 20220265865
- Publication date Aug 25, 2022
- Decibel Therapeutics, Inc.
- Joseph BURNS
- C07 - ORGANIC CHEMISTRY
Information Patent Application
ADENO-ASSOCIATED VIRUS VECTOR DELIVERY OF CYSTATHIONINE BETA-SYNTHA...
- Publication number 20220226502
- Publication date Jul 21, 2022
- Institute For Cancer Research d/b/aThe Research Institute of Fox Chase Cancer...
- Warren Kruger
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
COMPOSITIONS AND METHODS FOR TREATMENT OF HEMOCHROMATOSIS
- Publication number 20220220189
- Publication date Jul 14, 2022
- ULTRAGENYX PHARMACEUTICAL INC.
- Sean Christopher DAUGHERTY
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
Information Patent Application
COMPOUNDS AND METHODS FOR REDUCING ATXN3 EXPRESSION
- Publication number 20220195431
- Publication date Jun 23, 2022
- Ionis Pharmaceuticals, Inc.
- Hien Thuy Zhao
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
Information Patent Application
HUMANIZED VIPR2 COPY NUMBER VARIANT TRANSGENIC MOUSE MODEL FOR ANTI...
- Publication number 20220090124
- Publication date Mar 24, 2022
- Board of Supervisors of Louisiana State University and Agricultural and Mecha...
- Xiaohong LU
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...

Animal model for genetic diseases

Industry

Parent Industries

Current Industry

Patents Grantslast 30 patents

Transgenic animal having CREBBP gene into which mutation has been i...

Non-human animals models of retinoschisis

Treatment for autistic spectrum disorder (ASD) and relevant symptoms

Antisense compounds targeting genes associated with cystic fibrosis

Cell therapy with lentiviral transduced CSF2RA transgene in the tre...

Non-human animals comprising a humanized TTR locus and methods of use

Drug for retinal degenerative disease associated with photoreceptor...

Rat model of down syndrome and method for producing same

Methods of treating muscular dystrophy

Mutations in rhodopsin gene in zebrafish and uses thereof

Methods and compositions for treating chronic granulomatous disease

Fibrillin-1 mutations for modeling neonatal progeroid syndrome with...

Prevention and/or treatment of hearing loss or impairment

Methods and compositions for modifying a mutant dystrophin gene in...

Method of making a rat model of retinal degeneration and rat model...

Methods and compositions for modified factor IX fusion proteins

Optimized mini-dystrophin genes and expression cassettes and their use

Transcription modulation in animals using CRISPR/Cas systems

Use and production of CHD8+/− transgenic animals with behavioral ph...

Anti-factor D antibodies and uses thereof

Compositions and methods for enhanced genome editing

Genetically modified mouse with an inducible ACVR1 gene with a muta...

Methods of reducing FUS/TLS- or TDP-43-mediated neuronal cytotoxici...

Large animal model for developing therapeutic agents to treat impai...

Targeted RNA knockdown and knockout by type III-A Csm complexes

Disease model pig exhibiting stable phenotype, and production metho...

Delivery, use and therapeutic applications of the CRISPR-Cas system...

Antisense compounds targeting genes associated with cystic fibrosis

Methods for the treatment of Danon disease and other disorders of a...

Non-human animal models of retinoschisis

Patents Applicationslast 30 patents

HUMANIZED VIPR2 COPY NUMBER VARIANT TRANSGENIC MOUSE MODEL FOR ANTI...

IMMUNOTHERAPY COMPOSITIONS AND METHODS FOR TREATMENT OF TAUOPATHY A...

METHODS OF TREATMENT WITH AMINOLEVULINIC ACID SYNTHASE 2 (ALAS2) MO...

Use Of Citicoline In Regulating Respiratory Chain Function Related...

GENE THERAPY CONSTRUCTS AND METHODS FOR TREATMENT OF HEARING LOSS

MOUSE MODEL AND TREATMENT OF HEREDITARY INCLUSION BODY MYOPATHY

IMMUNOTHERAPY COMPOSITIONS AND METHODS FOR TREATMENT OF TAUOPATHY A...

Methods of Treating Muscular Dystrophy

Methods and Compositions for Modifying a Mutant Dystrophin Gene in...

DRUG FOR RETINAL DEGENERATIVE DISEASE ASSOCIATED WITH PHOTORECEPTOR...

PREVENTION AND/OR TREATMENT OF HEARING LOSS OR IMPAIRMENT

OPTIMIZED MINI-DYSTROPHIN GENES AND EXPRESSION CASSETTES AND THEIR USE

A HIGH-THROUGHPUT SCREENING METHOD TO DISCOVER OPTIMAL GRNA PAIRS F...

NOVEL METHOD FOR PREPARING ANIMAL MODEL OF CEREBROVASCULAR DISEASE...

GENE THERAPY FOR NIEMANN-PICK DISEASE TYPE A

GENETICALLY MODIFIED NON-HUMAN ANIMAL WITH HUMAN OR CHIMERIC CD38

TRANSCRIPTION MODULATION IN ANIMALS USING CRISPR/CAS SYSTEMS

METHODS OF CONTROLLING BODYWEIGHT BY MODULATING PHOSPHATIDYLINOSITO...

NON-HUMAN ANIMALS COMPRISING A HUMANIZED TTR LOCUS AND METHODS OF USE

TREATMENT OF MUCOPOLYSACCHARIDOSIS IVA

TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS

ANTI-FACTOR D ANTIBODIES AND USES THEREOF

COMPOSITIONS AND METHODS FOR GENE REPLACEMENT

TRANSGENIC NON-HUMAN ANIMAL CAPABLE OF CONTROLLING EXPRESSION OF TR...

Genetically Modified Non-Human Animals and Methods of Use Thereof

COMPOSITIONS AND METHODS FOR TREATING SENSORINEURAL HEARING LOSS US...

ADENO-ASSOCIATED VIRUS VECTOR DELIVERY OF CYSTATHIONINE BETA-SYNTHA...

COMPOSITIONS AND METHODS FOR TREATMENT OF HEMOCHROMATOSIS

COMPOUNDS AND METHODS FOR REDUCING ATXN3 EXPRESSION

HUMANIZED VIPR2 COPY NUMBER VARIANT TRANSGENIC MOUSE MODEL FOR ANTI...