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BIOMARKER RESEARCH FOR COL6-RELATED DYSTROPHIES

BIOMARKER RESEARCH FOR COL6-RELATED DYSTROPHIES

Search for reliable biomarkers that allow evaluating the progression of the disease and the response to new treatments.

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PERSONALIZED MEDICINE THERAPIES FOR CONGENITAL MUSCULAR DYSTROPHIES

PERSONALIZED MEDICINE THERAPIES FOR CONGENITAL MUSCULAR DYSTROPHIES

Development of advanced molecular diagnostic methods for the accurate diagnosis of congenital muscular dystrophies and novel gene therapies for the treatment of collagen VI deficiency.

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DEVELOPING TWO RNA DIRECTED APPROACHES TO TREATING THE RECURRENT GLYCINE VARIANTS P.G284R AND P.G293R IN COL6A1

DEVELOPING TWO RNA DIRECTED APPROACHES TO TREATING THE RECURRENT GLYCINE VARIANTS P.G284R AND P.G293R IN COL6A1

Chemical engineering of therapeutic siRNAs and use of circular RNA guides.

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EVALUATION AND DEVELOPMENT OF PERSONALISED THERAPIES FOR COLLAGEN VI-RELATED MYOPHATIES

EVALUATION AND DEVELOPMENT OF PERSONALISED THERAPIES FOR COLLAGEN VI-RELATED MYOPHATIES

Develop and validate an evaluation method and design and evaluate different nucleic acid therapies.

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CLOSING THE THERAPEUTIC GAP FOR CONGENITAL MUSCULAR DYSTROPHY

CLOSING THE THERAPEUTIC GAP FOR CONGENITAL MUSCULAR DYSTROPHY

Improve the Efficacy, Delivery, and Safety of CRISPR/Cas9 and Antisense Oligonucleotide-Based Therapies.

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NEURO-PSYCHOSOCIAL INTERVENTION FOR MINORS WITH NEUROMUSCULAR DISEASES

NEURO-PSYCHOSOCIAL INTERVENTION FOR MINORS WITH NEUROMUSCULAR DISEASES

Academic collaboration agreement between Fundación Noelia and Deusto Univerity to work in the psychosocial field of Col6 affected.

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OPTIMIZATION OF VIRAL VECTORS FOR THE EFFECTIVE GENE DELIVERY TO MUSCLE TISSUE CELLS

OPTIMIZATION OF VIRAL VECTORS FOR THE EFFECTIVE GENE DELIVERY TO MUSCLE TISSUE CELLS

Directed evolution of adeno-associated virus (AAV) capsids for effective gene delivery to the muscle fibro-adipogenic progenitors (FAPS)

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A NEW NANOPARTICLE-BASED DELIVERY SYSTEM FOR NUCLEIC ACIDS TO TREAT COLLAGEN VI-CMD

A NEW NANOPARTICLE-BASED DELIVERY SYSTEM FOR NUCLEIC ACIDS TO TREAT COLLAGEN VI-CMD

DYSTRO-SMARTY: a non-liposomal nanovesicle platform for delivery of nucleic acids to treat Collagen VI-related Congenital Muscular Dystrophy

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PERSONALIZED MEDICINE FOR COLLAGEN VI CONGENITAL MUSCULAR DYSTROPHIES

PERSONALIZED MEDICINE FOR COLLAGEN VI CONGENITAL MUSCULAR DYSTROPHIES

Congenital Muscular Dystrophies are a group of rare, highly disabling neuromuscular diseases with a reduced life expectancy.

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UPDATE OF THE INTERNATIONAL PATIENT REGISTRY OF CONGENITAL MUSCULAR DYSTROPHIES (CMDIR)

UPDATE OF THE INTERNATIONAL PATIENT REGISTRY OF CONGENITAL MUSCULAR DYSTROPHIES (CMDIR)

The International Registry of Congenital Muscular Dystrophies (CMDIR) was created in 2009 by CureCMD, a patient association of the United States, in collaboration with…

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RNA EDITING BY RECRUITING ENDOGENOUS ADAR USING LONG RNAS TO CORRECT GLYCINE SUBSTITUTIONS IN COL6-RD

RNA EDITING BY RECRUITING ENDOGENOUS ADAR USING LONG RNAS TO CORRECT GLYCINE SUBSTITUTIONS IN COL6-RD

Collagen VI-related dystrophies (COL6-RD) are commonly caused by dominant-negative pathogenic variants in the COL6A1,COL6A2 and COL6A3 genes…

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STUDY OF GENE THERAPY BY “EXON SKIPPING” FOR INTRONIC MUTATIONS IN THE PRE-CLINICAL PHASE

STUDY OF GENE THERAPY BY “EXON SKIPPING” FOR INTRONIC MUTATIONS IN THE PRE-CLINICAL PHASE

A large international research group named “Exon skipping, Intron 11” emerged to try to solve the diagnosis and promote research on finding a cure for a type of mutation…

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DEVELOPMENT OF SELECTIVE SILENCING MUTATION AS A THERAPY FOR THE COLLAGEN VI RELATED DYSTROPHIES

DEVELOPMENT OF SELECTIVE SILENCING MUTATION AS A THERAPY FOR THE COLLAGEN VI RELATED DYSTROPHIES

The project is based on gene silencing, a technique used to mask dominant mutations known in advance…

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NATURAL HISTORY, DIAGNOSIS AND TREATMENT OF COLLAGEN VI-RELATED MUSCULAR DYSTROPHIES

NATURAL HISTORY, DIAGNOSIS AND TREATMENT OF COLLAGEN VI-RELATED MUSCULAR DYSTROPHIES

nvestigating the application of gene editing technology to dominant mutations in collagen-VI genes…

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