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2025
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Invention
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Acid-alpha glucosidase variants and uses thereof.
The present invention relates to variants of a... |
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2024
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Invention
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Hybrid regulatory elements.
The present invention relates to hybrid transcription regulatory ele... |
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Invention
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Methods for aav vector re-administration. The invention relates to a combination of a recombinant... |
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Invention
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Prediction of multiple aav properties by deep learning. The invention relates to a method impleme... |
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Invention
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Fluidic system for producing extracellular vesicles and related method.
A fluidic system for pro... |
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Invention
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Novel optimized utrophin micro-genes. The present invention concerns a micro-utrophin comprising ... |
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Invention
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Novel mididystrophins. The present invention concerns mididystrophins comprising at least the rod... |
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Invention
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N-terminal truncated glycogen debranching enzymes for the treatment of glycogen storage disease i... |
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Invention
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Hybrid recombinant adeno-associated virus serotype between aav9 and aavrh74 with reduced liver tr... |
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Invention
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Lentiviral vector for dopamine synthesis. The invention relates to a lentiviral vector comprising... |
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Invention
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Machine learning method for phenotype prediction. The present invention relates to the generation... |
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Invention
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Induction of immune tolerance by aav vector comprising the combination of a liver detargeted caps... |
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2023
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Invention
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Lsd1 inhibitor and prmt6 inhibitor for use in the treatment of a disease associated with gain-of-... |
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Invention
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Tissue imaging methods and systems. The invention relates to a method for predicting treatment ef... |
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G/S
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Produits pharmaceutiques, vétérinaires et hygiéniques; préparations chimiques à usage médical, ph... |
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G/S
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Pharmaceutical, veterinary products, namely gene and cell therapy products for the treatment of d... |
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G/S
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Pharmaceutical, veterinary and hygienic products; chemical
preparations for medical and pharmace... |
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Invention
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Hybrid aav vector enhancing transgene expression in the liver. The invention relates to a hybrid ... |
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Invention
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Immunotherapy of skeletal myopathies using anti-fap car-t cells. The invention relates to an immu... |
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Invention
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Cns gene delivery using peripheral administration of aav vectors. The present invention relates t... |
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Invention
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Treatment of hyperbilirubinemia.
The invention relates to a nucleic acid sequence useful in the ... |
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Invention
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N-terminal truncated gde for the treatment of glycogen storage disease iii. The present invention... |
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Invention
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Peptide-modified aav capsid with enhanced muscle transduction efficiency. The invention relates t... |
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Invention
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Use of bazedoxifene for increasing muscle survival. The invention relates to the use of bazedoxif... |
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Invention
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Novel polynucleotides encoding a human fkrp protein.
The present invention concerns synthetic po... |
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2022
|
Invention
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Gamma-sarcoglycan gene transfer increase using modified itr sequences. The present invention rela... |
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Invention
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Calpain-3 gene transfer increase using modified itr sequences. The present invention relates to a... |
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Invention
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Alpha-sarcoglycan gene transfer increase using modified itr sequences. The present invention rela... |
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Invention
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Fukutin related protein gene transfer increase using modified itr sequences. The present inventio... |
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Invention
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Treatment of glycogen storage disease iii.
The present invention relates to vectors and composit... |
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Invention
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Stable pseudotyped lentiviral particles and uses thereof.
The present invention relates to a met... |
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Invention
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Hybrid promoters for gene expression in muscles and in the cns.
The present invention relates to... |
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Invention
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Compositions and methods for increasing or enhancing transduction of gene therapy vectors and for... |
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|
Invention
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Compositions and methods for improving viral vector efficiency.
The present invention relates to... |
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|
Invention
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Use of bazedoxifene for increasing muscle survival.
The invention concerns the use of bazedoxife... |
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2021
|
Invention
|
Lysosomal acid lipase variants and uses thereof.
The present invention relates to variants of ly... |
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Invention
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Enhancing utrophin expression in cell by inducing mutations within utrophin regulatory elements a... |
|
|
Invention
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Peptide-modified aav capsid.
The invention relates to a peptide-modified AAV capsid with improve... |
|
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Invention
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C-terminal truncated gde for the treatment of glycogen storage disease iii.
The present inventio... |
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Invention
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A novel muscle-specific promoter.
The present invention concerns a novel short promoter characte... |
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|
Invention
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Method for engineering novel hybrid aav capsids through hyper variable regions swapping.
The inv... |
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Invention
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Gene therapy expression system allowing an adequate expression in the muscles and in the heart of... |
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|
Invention
|
Treatment of dilated cardiomyopathies.
The invention relates to the treatment of dilated cardiom... |
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|
Invention
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Treatment of genetic dilated cardiomyopathies.
The invention relates to the treatment of genetic... |
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|
Invention
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Nrf2 activator for use in treating dilated cardiomyopathies.
The present invention relates to th... |
|
|
Invention
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Cilp-1 inhibitors for use in the treatment of dilated cardiomyopathies.
The present disclosure r... |
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|
Invention
|
Vectors encoding a glucose-6-phosphatase (g6pase-a) for gene therapy.
The invention relates to a... |
|
|
Invention
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Use of a synthetic aav capsid for gene therapy of muscle and central nervous system disorders.
T... |
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|
Invention
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Methods for preventing induction of immune responses to the transduced cells expressing a transge... |
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|
Invention
|
Vectors for the treatment of acid ceramidase deficiency.
The present invention relates to a vect... |
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2020
|
Invention
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Combination of a proteasome inhibitor and a hdac inhibitor and its use for the treatment of genet... |
|
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Invention
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Gene therapy expression system alleviating cardiac toxicity of fkrp.
The present invention conce... |
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|
Invention
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Gene therapy of fibroblast growth factor 23 related hypophosphatemic diseases.
The invention rel... |
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2012
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G/S
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Biological and chemical products used in the pharmaceutical and life sciences field, namely, reag... |
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G/S
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Pharmaceutical preparations for medical purposes,
pharmaceutical or chemical products, chemical-... |
