{"id":19389,"date":"2022-04-04T10:23:04","date_gmt":"2022-04-04T10:23:04","guid":{"rendered":"https:\/\/fundacionnoelia.org\/proyectos-de-investigacion\/"},"modified":"2025-02-26T11:02:58","modified_gmt":"2025-02-26T11:02:58","slug":"research-projects","status":"publish","type":"page","link":"https:\/\/fundacionnoelia.org\/en\/research-projects\/","title":{"rendered":"Research Projects"},"content":{"rendered":"[vc_row css=”.vc_custom_1647264715748{margin-top: 0px !important;border-top-width: 0px !important;padding-top: 0px !important;background-color: #951b81 !important;}”][vc_column][vc_empty_space height=”20px”][vc_row_inner][vc_column_inner width=”1\/4″]
0<\/span><\/div>
Years<\/div><\/div><\/div><\/div>[\/vc_column_inner][vc_column_inner width=”1\/4″]
0<\/span><\/div>
Projects<\/div><\/div><\/div><\/div>[\/vc_column_inner][vc_column_inner width=”1\/4″]
0<\/span>\u20ac<\/span><\/div>
Invested<\/div><\/div><\/div><\/div>[\/vc_column_inner][vc_column_inner width=”1\/4″]
0<\/span>%<\/span><\/div>
Implication<\/div><\/div><\/div><\/div>[\/vc_column_inner][\/vc_row_inner][\/vc_column][\/vc_row][vc_row][vc_column][vc_empty_space][\/vc_column][\/vc_row][vc_row][vc_column width=”1\/6″][\/vc_column][vc_column width=”4\/6″][vc_row_inner][vc_column_inner width=”1\/3″]
\"INTERNATIONAL<\/figure>

INTERNATIONAL CONSORTIUM FOR COL6-CMD RESEARCH<\/h2>

Grant Call for Start-up Costs: International Consortium for Col6-CMD Research<\/p>\n

View Project<\/a><\/p>\n<\/div><\/div><\/div>[\/vc_column_inner][vc_column_inner width=”1\/3″]

\"PERSONALIZED<\/figure>

PERSONALIZED MEDICINE THERAPIES FOR CONGENITAL MUSCULAR DYSTROPHIES<\/h2>

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.<\/p>\n

View Project<\/a><\/p>\n<\/div><\/div><\/div>[\/vc_column_inner][vc_column_inner width=”1\/3″]

\"DEVELOPING<\/figure>

DEVELOPING TWO RNA DIRECTED APPROACHES TO TREATING THE RECURRENT GLYCINE VARIANTS P.G284R AND P.G293R IN COL6A1<\/h2>

Chemical engineering of therapeutic siRNAs and use of circular RNA guides.<\/p>\n

View Project<\/a><\/p>\n<\/div><\/div><\/div>[\/vc_column_inner][\/vc_row_inner][\/vc_column][vc_column width=”1\/6″][\/vc_column][\/vc_row][vc_row][vc_column width=”1\/6″][\/vc_column][vc_column width=”4\/6″][vc_row_inner][vc_column_inner width=”1\/3″]

\"EVALUATION<\/figure>

EVALUATION AND DEVELOPMENT OF PERSONALISED THERAPIES FOR COLLAGEN VI-RELATED MYOPHATIES<\/h2>

Develop and validate an evaluation method and design and evaluate different nucleic acid therapies.<\/p>\n

View Project<\/a><\/p>\n<\/div><\/div><\/div>[\/vc_column_inner][vc_column_inner width=”1\/3″]

\"EVALUATION<\/figure>

EVALUATION AND DEVELOPMENT OF PERSONALISED THERAPIES FOR COLLAGEN VI-RELATED MYOPHATIES<\/h2>

Develop and validate an evaluation method and design and evaluate different nucleic acid therapies.<\/p>\n

View Project<\/a><\/p>\n<\/div><\/div><\/div>[\/vc_column_inner][vc_column_inner width=”1\/3″]

\"CLOSING<\/figure>

CLOSING THE THERAPEUTIC GAP FOR CONGENITAL MUSCULAR DYSTROPHY<\/h2>

Improve the Efficacy, Delivery, and Safety of CRISPR\/Cas9 and Antisense Oligonucleotide-Based Therapies.<\/p>\n

View Project<\/a><\/p>\n<\/div><\/div><\/div>[\/vc_column_inner][\/vc_row_inner][\/vc_column][vc_column width=”1\/6″][\/vc_column][\/vc_row][vc_row][vc_column width=”1\/6″][\/vc_column][vc_column width=”4\/6″][vc_row_inner][vc_column_inner width=”1\/3″]

\"NEURO-PSYCHOSOCIAL<\/figure>

NEURO-PSYCHOSOCIAL INTERVENTION FOR MINORS WITH NEUROMUSCULAR DISEASES<\/h2>

Academic collaboration agreement between Fundaci\u00f3n Noelia and Deusto Univerity to work in the psychosocial field of Col6 affected.<\/p>\n

View Project<\/a><\/p>\n<\/div><\/div><\/div>[\/vc_column_inner][vc_column_inner width=”1\/3″]

\"OPTIMIZATION<\/figure>

OPTIMIZATION OF VIRAL VECTORS FOR THE EFFECTIVE GENE DELIVERY TO MUSCLE TISSUE CELLS<\/h2>

Directed evolution of adeno-associated virus (AAV) capsids for effective gene delivery to the muscle fibro-adipogenic progenitors (FAPS)<\/p>\n

View Project<\/a><\/p>\n<\/div><\/div><\/div>[\/vc_column_inner][vc_column_inner width=”1\/3″]

\"A<\/figure>

A NEW NANOPARTICLE-BASED DELIVERY SYSTEM FOR NUCLEIC ACIDS TO TREAT COLLAGEN VI-CMD<\/h2>

DYSTRO-SMARTY: a non-liposomal nanovesicle platform for delivery of nucleic acids to treat Collagen VI-related Congenital Muscular Dystrophy<\/p>\n

View Project<\/a><\/p>\n<\/div><\/div><\/div>[\/vc_column_inner][\/vc_row_inner][\/vc_column][vc_column width=”1\/6″][\/vc_column][\/vc_row][vc_row][vc_column width=”1\/6″][\/vc_column][vc_column width=”4\/6″][vc_row_inner][vc_column_inner width=”1\/3″]

\"PERSONALIZED<\/figure>

PERSONALIZED MEDICINE FOR COLLAGEN VI CONGENITAL MUSCULAR DYSTROPHIES<\/h2>

Congenital Muscular Dystrophies are a group of rare, highly disabling neuromuscular diseases with a reduced life expectancy.<\/p>\n

View Project<\/a><\/p>\n<\/div><\/div><\/div>[\/vc_column_inner][vc_column_inner width=”1\/3″]

\"UPDATE<\/figure>

UPDATE OF THE INTERNATIONAL PATIENT REGISTRY OF CONGENITAL MUSCULAR DYSTROPHIES (CMDIR)<\/h2>

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

View Project<\/a><\/p>\n<\/div><\/div><\/div>[\/vc_column_inner][vc_column_inner width=”1\/3″]

\"RNA<\/figure>

RNA EDITING BY RECRUITING ENDOGENOUS ADAR USING LONG RNAS TO CORRECT GLYCINE SUBSTITUTIONS IN COL6-RD<\/h2>

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

View Project<\/a><\/p>\n<\/div><\/div><\/div>[\/vc_column_inner][\/vc_row_inner][\/vc_column][vc_column width=”1\/6″][\/vc_column][\/vc_row][vc_row][vc_column width=”1\/6″][\/vc_column][vc_column width=”4\/6″][vc_row_inner][vc_column_inner width=”1\/3″]

\"STUDY<\/figure>

STUDY OF GENE THERAPY BY \u201cEXON SKIPPING\u201d FOR INTRONIC MUTATIONS IN THE PRE-CLINICAL PHASE<\/h2>

A large international research group named \u201cExon skipping, Intron 11\u201d emerged to try to solve the diagnosis and promote research on finding a cure for a type of mutation…<\/p>\n

View Project<\/a><\/p>\n<\/div><\/div><\/div>[\/vc_column_inner][vc_column_inner width=”1\/3″]

\"DEVELOPMENT<\/figure>

DEVELOPMENT OF SELECTIVE SILENCING MUTATION AS A THERAPY FOR THE COLLAGEN VI RELATED DYSTROPHIES<\/h2>

The project is based on gene silencing, a technique used to mask dominant mutations known in advance…<\/p>\n

View Project<\/a><\/p>\n<\/div><\/div><\/div>[\/vc_column_inner][vc_column_inner width=”1\/3″]

\"NATURAL<\/figure>

NATURAL HISTORY, DIAGNOSIS AND TREATMENT OF COLLAGEN VI-RELATED MUSCULAR DYSTROPHIES<\/h2>

nvestigating the application of gene editing technology to dominant mutations in collagen-VI genes…<\/p>\n

View Project<\/a><\/p>\n<\/div><\/div><\/div>[\/vc_column_inner][\/vc_row_inner][\/vc_column][vc_column width=”1\/6″][\/vc_column][\/vc_row]\n","protected":false},"excerpt":{"rendered":"

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