regulating RNA stability, not being an intron

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CPC
C12N2830/50

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CCHEMISTRY METALLURGY C12Biochemistry C12NMICRO-ORGANISMS OR ENZYMES COMPOSITIONS THEREOF PROPAGATING, PRESERVING OR MAINTAINING MICRO-ORGANISMS MUTATION OR GENETIC ENGINEERING CULTURE MEDIA C12N2830/00Vector systems having a special element relevant for transcription

Current Industry

C12N2830/50regulating RNA stability, not being an intron

Industries

Patents Grantslast 30 patents

Information Patent Grant
Lassa virus vaccine
- Patent number 12,161,711
- Issue date Dec 10, 2024
- CureVac SE
- Edith Jasny
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Optimized expression cassettes for gene therapy
- Patent number 12,151,001
- Issue date Nov 26, 2024
- Tenaya Therapeutics, Inc.
- Huanyu Zhou
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
CRISPR therapy
- Patent number 12,152,242
- Issue date Nov 26, 2024
- The Curators of the University of Missouri
- Dongsheng Duan
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Therapy
- Patent number 12,121,593
- Issue date Oct 22, 2024
- The University of Bristol
- Moin Ahson Saleem-Uddin
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Self-replicating RNA and uses thereof
- Patent number 12,115,217
- Issue date Oct 15, 2024
- TRUSTEES OF BOSTON UNIVERSITY
- Joshua Edward McGee
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Adeno-associated virus vectors for treatment of Rett syndrome
- Patent number 12,104,163
- Issue date Oct 1, 2024
- Sarepta Therapeutics, Inc.
- Colin O'Banion
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Modified adenoviruses
- Patent number 12,098,383
- Issue date Sep 24, 2024
- GRITSTONE BIO, INC.
- Karin Jooss
- C07 - ORGANIC CHEMISTRY
Information Patent Grant
Codon-optimized acid alpha-glucosidase expression cassettes and met...
- Patent number 12,084,693
- Issue date Sep 10, 2024
- SPARK THERAPEUTICS, INC.
- Xavier Anguela
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Transgene cassettes, AAV vectors and AAV viral vectors for the expr...
- Patent number 12,077,772
- Issue date Sep 3, 2024
- The Board of Regents of the University of Texas System
- Steven J. Gray
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Adeno-associated virus compositions for restoring PAH gene function...
- Patent number 12,076,420
- Issue date Sep 3, 2024
- HOMOLOGY MEDICINES, INC.
- Jason Boke Wright
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Stabilization of poly(A) sequence encoding DNA sequences
- Patent number 12,054,723
- Issue date Aug 6, 2024
- BIONTECH SE
- Florian Eberle
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Synthetic genetic elements for biomanufacture
- Patent number 12,049,638
- Issue date Jul 30, 2024
- Janssen Biotech, Inc.
- William Lloyd Perry
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
BCMA chimeric antigen receptors
- Patent number 12,006,369
- Issue date Jun 11, 2024
- 2seventy bio, Inc.
- Richard Morgan
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Gene therapies for lysosomal disorders
- Patent number 11,993,790
- Issue date May 28, 2024
- Prevail Therapeutics, Inc.
- Asa Abeliovich
- C07 - ORGANIC CHEMISTRY
Information Patent Grant
AADC polynucleotides for the treatment of Parkinson's disease
- Patent number 11,975,056
- Issue date May 7, 2024
- VOYAGER THERAPEUTICS, INC.
- Robert Kotin
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Gene therapy for tuberous sclerosis
- Patent number 11,958,887
- Issue date Apr 16, 2024
- The General Hospital Corporation
- Xandra Breakefield
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Methods of treating phenylketonuria
- Patent number 11,952,585
- Issue date Apr 9, 2024
- HOMOLOGY MEDICINES, INC.
- Omar L. Francone
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Means and methods for improved regeneration
- Patent number 11,939,586
- Issue date Mar 26, 2024
- VIB VZW
- Lieven De Veylder
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Methods and materials for NT-3 gene therapy
- Patent number 11,926,653
- Issue date Mar 12, 2024
- Research Institute at Nationwide Children's Hospital
- Zarife Sahenk
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Adeno-associated viral vectors for treatment of Niemann-Pick Diseas...
- Patent number 11,905,523
- Issue date Feb 20, 2024
- Ginkgo BioWorks, Inc.
- Daniel McCoy
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Recombinant herpesvirus of turkey vectors expressing antigens of av...
- Patent number 11,883,481
- Issue date Jan 30, 2024
- Zoetis Services LLC
- Sing Rong
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Bicistronic AAV vector for RNA interference in ALS
- Patent number 11,872,290
- Issue date Jan 16, 2024
- Ecole Polytechnique Federale de Lausanne (EPFL)
- Julianne Aebischer
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Vector-mediated immune tolerance in the eye
- Patent number 11,866,480
- Issue date Jan 9, 2024
- The University of North Carolina at Chapel Hill
- Matthew Louis Hirsch
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Compositions and methods for enhanced gene expression
- Patent number 11,773,406
- Issue date Oct 3, 2023
- Adverum Biotechnologies, Inc.
- Annahita Keravala
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Synthetic DNA vectors and methods of use
- Patent number 11,766,490
- Issue date Sep 26, 2023
- INTERGALACTIC THERAPEUTICS, INC.
- Bruce C. Schnepp
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Compositions and methods for inducing immune responses
- Patent number 11,759,515
- Issue date Sep 19, 2023
- Arcturus Therapeutics, Inc.
- Sean Michael Sullivan
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Artificial nucleic acid molecules for improved protein expression
- Patent number 11,761,009
- Issue date Sep 19, 2023
- CureVac SE
- Andreas Thess
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Recombinant adeno-associated virus vectors with CD14 promoter and u...
- Patent number 11,761,020
- Issue date Sep 19, 2023
- Aavocyte, Inc.
- Aiquan Chang
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Neuroreceptor compositions and methods of use
- Patent number 11,760,788
- Issue date Sep 19, 2023
- Pathways Neuro Pharma, Inc.
- Warren C. Lau
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Coronavirus vaccine compositions and methods
- Patent number 11,744,887
- Issue date Sep 5, 2023
- Arcturus Therapeutics, Inc.
- Sean Michael Sullivan
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE

Patents Applicationslast 30 patents

Information Patent Application
COMPOSITIONS AND METHODS FOR TREATING A MUSCULAR DYSTROPHY
- Publication number 20250019721
- Publication date Jan 16, 2025
- ULTRAGENYX PHARMACEUTICAL INC.
- Matthew Scott Fuller
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
SELF-REPLICATING RNA AND USES THEREOF
- Publication number 20250018028
- Publication date Jan 16, 2025
- Trustees of Boston University
- Joshua Edward MCGEE
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
POLYNUCLEOTIDE COMPOSITIONS, RELATED FORMULATIONS, AND METHODS OF U...
- Publication number 20250019409
- Publication date Jan 16, 2025
- ReCode Therapeutics, Inc.
- Mirko Hennig
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ANTISENSE TRANSFER VECTORS AND METHODS OF USE THEREOF
- Publication number 20250009802
- Publication date Jan 9, 2025
- LUDWIG INSTITUTE FOR CANCER RESEARCH LTD.
- Melita Irving
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
NOVEL 5'-UNTRANSLATED REGION ELEMENT AND USE THEREOF
- Publication number 20250011770
- Publication date Jan 9, 2025
- THEMEDIUM THERAPEUTICS CO., LTD
- Tao KANG
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
STABILIZATION OF POLY(A) SEQUENCE ENCODING DNA SEQUENCES
- Publication number 20250011797
- Publication date Jan 9, 2025
- BIONTECH SE
- Florian Eberle
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
GENE THERAPY FOR SPINAL MUSCULAR ATROPHY
- Publication number 20250011812
- Publication date Jan 9, 2025
- University of Massachusetts
- Jun Xie
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
GENE THERAPY FOR GALACTOSEMIA
- Publication number 20250001008
- Publication date Jan 2, 2025
- BridgeBio Gene Therapy Research, Inc.
- Clayton BEARD
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
AAV CAPSID VARIANTS AND USES THEREOF
- Publication number 20250001012
- Publication date Jan 2, 2025
- Mathieu Emmanuel Nonnenmacher
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Vectors for Increasing NPRL2 Expression in Cancer Cells and Methods...
- Publication number 20250002546
- Publication date Jan 2, 2025
- Genprex, Inc.
- Michael Redman
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
RETGC Gene Therapy
- Publication number 20250002936
- Publication date Jan 2, 2025
- MEIRAGTX UK II LIMITED
- Anastasios Georgiadis
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
NOVEL METHODS OF USING ADENO-ASSOCIATED VIRUS 11 (AAV11)
- Publication number 20240424085
- Publication date Dec 26, 2024
- Massachusetts Eye and Ear Infirmary
- Luc H. Vandenberghe
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
AAV PARTICLES COMPRISING A LIVER-TROPIC CAPSID PROTEIN AND ALPHA-GA...
- Publication number 20240424141
- Publication date Dec 26, 2024
- CANbridge Pharmaceuticals, Inc.
- Yunxiang Zhu
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
COMPOSITIONS AND METHODS FOR TREATING INTERLEUKIN 7 RECEPTOR DEFICI...
- Publication number 20240417444
- Publication date Dec 19, 2024
- The Children's Hospital of Philadelphia
- Stefano Rivella
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
AADC POLYNUCLEOTIDES FOR THE TREATMENT OF PARKINSON'S DISEASE
- Publication number 20240415943
- Publication date Dec 19, 2024
- VOYAGER THERAPEUTICS, INC.
- Robert Kotin
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
BCMA CHIMERIC ANTIGEN RECEPTORS
- Publication number 20240417481
- Publication date Dec 19, 2024
- 2seventy bio, Inc.
- Richard Morgan
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
NUCLEIC ACID CONSTRUCTS, VIRAL VECTORS AND VIRAL PARTICLES
- Publication number 20240417434
- Publication date Dec 19, 2024
- UCB Biopharma SRL
- Natalia RODRIGUEZ ALVAREZ
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
SYSTEMS AND METHODS FOR DETERMINING EFFECTS OF THERAPIES AND GENETI...
- Publication number 20240412818
- Publication date Dec 12, 2024
- DEEP GENOMICS INCORPORATED
- Brendan Frey
- G06 - COMPUTING CALCULATING COUNTING
Information Patent Application
TREATMENT OF HEREDITARY ANGIOEDEMA WITH AAV GENE THERAPY VECTORS AN...
- Publication number 20240408240
- Publication date Dec 12, 2024
- BioMarin Pharmaceutical Inc.
- Peter COLOSI
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS USEFUL FOR TREATMENT OF CHARCOT-MARIE-TOOTH DISEASE
- Publication number 20240401078
- Publication date Dec 5, 2024
- The Trustees of the University of Pennsylvania
- James M. Wilson
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ADENO-ASSOCIATED VIRUS VECTORS FOR TREATMENT OF RETT SYNDROME
- Publication number 20240392316
- Publication date Nov 28, 2024
- Sarepta Therapeutics, Inc.
- Colin O'Banion
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
POLY-TAILED AND POLY-CAPPED MRNA AND USES THEREOF
- Publication number 20240392311
- Publication date Nov 28, 2024
- THE BROAD INSTITUTE, INC.
- Xiao WANG
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
TARGETED GENE THERAPY FOR DM-1 MYOTONIC DYSTROPHY
- Publication number 20240384271
- Publication date Nov 21, 2024
- Genzyme Corporation
- Seng Cheng
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
GENE THERAPY CONSTRUCT FOR TREATING POMPE DISEASE, PHARMACEUTICAL C...
- Publication number 20240384297
- Publication date Nov 21, 2024
- BEIJING GENECRADLE PHARMACEUTICAL CO., LTD.
- Xiaobing WU
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHOD FOR TREATING A DISEASE
- Publication number 20240384245
- Publication date Nov 21, 2024
- SIRT6 LTD
- Hagit ASHUSH
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS AND MATERIALS FOR NT-3 GENE THERAPY
- Publication number 20240383956
- Publication date Nov 21, 2024
- Research Institute at Nationwide Children's Hospital
- Zarife Sahenk
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
TRANSGENE CASSETTES, AAV VECTORS AND AAV VIRAL VECTORS FOR THE EXPR...
- Publication number 20240376498
- Publication date Nov 14, 2024
- The Board of Regents of the University of Texas System
- Steven J. GRAY
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
RECOMBINANT HERPESVIRUS OF TURKEY VECTORS EXPRESSING ANTIGENS OF AV...
- Publication number 20240374702
- Publication date Nov 14, 2024
- Zoetis Services LLC
- Sing RONG
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS AND METHODS FOR TREATING LEBER'S HEREDITARY OPTIC NEUR...
- Publication number 20240376164
- Publication date Nov 14, 2024
- Wuhan Neurophth Biotechnology Limited Company
- Bin LI
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
POLYNUCLEOTIDES ENCODING URIDINE DIPHOSPHATE GLYCOSYLTRANSFERASE 1...
- Publication number 20240376445
- Publication date Nov 14, 2024
- ModernaTX, Inc.
- David Reid
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...

regulating RNA stability, not being an intron

Industry

Parent Industries

Current Industry

Patents Grantslast 30 patents

Lassa virus vaccine

Optimized expression cassettes for gene therapy

CRISPR therapy

Therapy

Self-replicating RNA and uses thereof

Adeno-associated virus vectors for treatment of Rett syndrome

Modified adenoviruses

Codon-optimized acid alpha-glucosidase expression cassettes and met...

Transgene cassettes, AAV vectors and AAV viral vectors for the expr...

Adeno-associated virus compositions for restoring PAH gene function...

Stabilization of poly(A) sequence encoding DNA sequences

Synthetic genetic elements for biomanufacture

BCMA chimeric antigen receptors

Gene therapies for lysosomal disorders

AADC polynucleotides for the treatment of Parkinson's disease

Gene therapy for tuberous sclerosis

Methods of treating phenylketonuria

Means and methods for improved regeneration

Methods and materials for NT-3 gene therapy

Adeno-associated viral vectors for treatment of Niemann-Pick Diseas...

Recombinant herpesvirus of turkey vectors expressing antigens of av...

Bicistronic AAV vector for RNA interference in ALS

Vector-mediated immune tolerance in the eye

Compositions and methods for enhanced gene expression

Synthetic DNA vectors and methods of use

Compositions and methods for inducing immune responses

Artificial nucleic acid molecules for improved protein expression

Recombinant adeno-associated virus vectors with CD14 promoter and u...

Neuroreceptor compositions and methods of use

Coronavirus vaccine compositions and methods

Patents Applicationslast 30 patents

COMPOSITIONS AND METHODS FOR TREATING A MUSCULAR DYSTROPHY

SELF-REPLICATING RNA AND USES THEREOF

POLYNUCLEOTIDE COMPOSITIONS, RELATED FORMULATIONS, AND METHODS OF U...

ANTISENSE TRANSFER VECTORS AND METHODS OF USE THEREOF

NOVEL 5'-UNTRANSLATED REGION ELEMENT AND USE THEREOF

STABILIZATION OF POLY(A) SEQUENCE ENCODING DNA SEQUENCES

GENE THERAPY FOR SPINAL MUSCULAR ATROPHY

GENE THERAPY FOR GALACTOSEMIA

AAV CAPSID VARIANTS AND USES THEREOF

Vectors for Increasing NPRL2 Expression in Cancer Cells and Methods...

RETGC Gene Therapy

NOVEL METHODS OF USING ADENO-ASSOCIATED VIRUS 11 (AAV11)

AAV PARTICLES COMPRISING A LIVER-TROPIC CAPSID PROTEIN AND ALPHA-GA...

COMPOSITIONS AND METHODS FOR TREATING INTERLEUKIN 7 RECEPTOR DEFICI...

AADC POLYNUCLEOTIDES FOR THE TREATMENT OF PARKINSON'S DISEASE

BCMA CHIMERIC ANTIGEN RECEPTORS

NUCLEIC ACID CONSTRUCTS, VIRAL VECTORS AND VIRAL PARTICLES

SYSTEMS AND METHODS FOR DETERMINING EFFECTS OF THERAPIES AND GENETI...

TREATMENT OF HEREDITARY ANGIOEDEMA WITH AAV GENE THERAPY VECTORS AN...

COMPOSITIONS USEFUL FOR TREATMENT OF CHARCOT-MARIE-TOOTH DISEASE

ADENO-ASSOCIATED VIRUS VECTORS FOR TREATMENT OF RETT SYNDROME

POLY-TAILED AND POLY-CAPPED MRNA AND USES THEREOF

TARGETED GENE THERAPY FOR DM-1 MYOTONIC DYSTROPHY

GENE THERAPY CONSTRUCT FOR TREATING POMPE DISEASE, PHARMACEUTICAL C...

METHOD FOR TREATING A DISEASE

METHODS AND MATERIALS FOR NT-3 GENE THERAPY

TRANSGENE CASSETTES, AAV VECTORS AND AAV VIRAL VECTORS FOR THE EXPR...

RECOMBINANT HERPESVIRUS OF TURKEY VECTORS EXPRESSING ANTIGENS OF AV...

COMPOSITIONS AND METHODS FOR TREATING LEBER'S HEREDITARY OPTIC NEUR...

POLYNUCLEOTIDES ENCODING URIDINE DIPHOSPHATE GLYCOSYLTRANSFERASE 1...