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2025
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G/S
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Pharmaceutical preparations for the treatment of neuromuscular diseases and disorders, muscular d... |
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G/S
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Providing information about medical and healthcare research and clinical trials; Providing inform... |
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G/S
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Pharmaceutical preparations for the treatment of
neuromuscular diseases and disorders, muscular ... |
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Invention
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Humanized umod mouse model generation, characterization and methods of use. hUMODhUMODhUMOD non-h... |
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Invention
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Exon skipping oligomers for muscular dystrophy.
Antisense oligomers complementary to a selected ... |
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Invention
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Processes for preparing phosphorodiamidate morpholino oligomers.
Provided herein are processes f... |
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2024
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Invention
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Trimeric peptides for antisense delivery.
Provided herein are oligonucleotides, trimeric peptide... |
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Invention
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Humanized dnm2 mouse model generation, characterization and methods of use. hDNM2 hDNM2hDNM2 non-... |
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Invention
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Antisense oligomers for treatment of centronuclear myopathies. Provided herein are oligonucleotid... |
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Invention
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Antisense oligomers and methods of using the same for treating diseases associated with the acid ... |
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Invention
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Adapted human dystrophins with enhanced actin-binding affinity for treating muscular dystrophies.... |
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G/S
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Viruses, viral vectors, and viral constructs for use in the manufacture of pharmaceuticals for th... |
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Invention
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Methods of risk assessment for muscular dystrophy gene therapy. The disclosure provides for metho... |
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Invention
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Exon skipping compositions for treating muscular dystrophy.
Antisense molecules capable of bindi... |
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Invention
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Modified antisense oligomers for exon inclusion in spinal muscular atrophy.
The present disclosu... |
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Invention
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Exon skipping oligomer conjugates for muscular dystrophy.
Antisense oligomer conjugates compleme... |
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Invention
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Cell-penetrating peptides for antisense delivery.
Provided herein are oligonucleotides, cell pen... |
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Invention
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Non-canonical cell-penetrating peptides for antisense oligomer delivery.
Provided herein are ant... |
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Invention
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Non-canonical cell-penetrating peptides for antisense oligomer delivery. Provided herein are anti... |
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Invention
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Multiple exon skipping compositions for dmd.
Provided are antisense molecules capable of binding... |
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Invention
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Processes for preparing phosphorodiamidate morpholino oligomers via fast-flow synthesis.
Provide... |
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Invention
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Exon skipping oligomer conjugates for muscular dystrophy.
Antisense oligomers complementary to a... |
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Invention
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Adeno-associated virus vectors for treatment of rett syndrome.
The disclosure provides nucleic a... |
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Invention
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Fusion proteins comprising cas enzymes and dna-binding proteins. Disclosed herein are fusion prot... |
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Invention
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Antisense-induced exon2 inclusion in acid alpha-glucosidase.
The present disclosure relates to a... |
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Invention
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Peptide oligonucleotide conjugates.
Provided herein are peptide-oligomer-conjugates. Also provid... |
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Invention
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Antisense oligomers for treatment of chronic kidney disease.
Provided herein are compounds such ... |
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Invention
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Antisense oligomers for treatment of chronic kidney disease. Provided herein are compounds such a... |
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Invention
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Systemic delivery of adeno-associated virus vector expressing gamma-sarcoglycan and the treatment... |
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Invention
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Exon skipping oligomers and oligomer conjugates for muscular dystrophy.
Antisense oligomers and ... |
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Invention
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Raav production methods. Disclosed herein are improved methods of production of recombinant adeno... |
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Invention
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Antisense-induced exon exclusion in type vii collagen.
The present disclosure relates to antisen... |
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2023
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Invention
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Formulation of an antisense oligomer conjugate. Provided herein are pharmaceutical compositions c... |
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Invention
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Pompe disease mouse model generation, characterization and methods of use. Disclosed herein are t... |
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Invention
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Recombinant aav vectors for treating muscular dystrophy. DMDDMD gene to determine if rAAV gene th... |
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Invention
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Dmd antisense oligonucleotide-mediated exon skipping efficiency. Provided herein are oligonucleot... |
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Invention
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Methods of treating muscular dystrophy. e.ge.g., an AAVrh74 vector, and an enzyme that cleaves Ig... |
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G/S
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Connecting healthcare providers with medical specialists and researchers to enable communication ... |
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G/S
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Pharmaceutical preparations for the treatment of muscular dystrophies, central nervous system dis... |
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Invention
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Oligonucleotides for treating expanded repeat diseases.
The invention provides for a method for ... |
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Invention
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Peptide oligonucleotide conjugates. Provided herein are oligonucleotides, peptides, and peptide-o... |
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Invention
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Phosphorodiamidate morpholino oligomer conjugates.
Novel antisense oligonucleotide conjugates th... |
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2022
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Invention
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Antisense oligomer compounds.
A modified antisense oligonucleotide of about 10 to about 40 nucle... |
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Invention
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Morpholino oligomers for treatment of peripheral myelin protein 22 related diseases.
Provided he... |
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Invention
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Antisense oligonucleotides having one or more abasic units.
Provided herein are oligonucleotides... |
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G/S
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Pharmaceutical preparations. Medical education services; Medical training and teaching. Medical s... |
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Invention
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Production of recombinant aav vectors for treating muscular dystrophy.
The present disclosure pr... |
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G/S
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Providing health information; Providing health information in the field of muscular dystrophy, Du... |
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2021
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Invention
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Compositions comprising exon skipping oligonucleotide conjugates for treating muscular dystrophy.... |
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Invention
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Suspension mode seed train development for adherent cells.
The disclosure is directed to a metho... |
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G/S
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Pharmaceutical preparations for the treatment of neuromuscular diseases and disorders |
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G/S
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Providing health information |
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G/S
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Pharmaceutical preparations featuring antisense oligonucleotides for the treatment of neuromuscul... |
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G/S
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Providing information about medical and healthcare research and clinical trials; providing inform... |
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Invention
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Designing antisense oligonucleotide delivery peptides by interpretable machine learning.
Provide... |
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G/S
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Pharmaceutical preparations featuring antisense oligonucleotides, for the treatment of neuromuscu... |
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2020
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Invention
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Aav transfer cassette.
Described herein are AAV transfer cassettes and plasmids used in the prod... |
