PROG HEAD — G1 — DOTT S3 — CRISPR/CAS9 AS A FUNCTIONAL ANALYSIS TECHNOLOGY — FN (Q1970834): Difference between revisions
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Project in Italy | Project Q1970834 in Italy | ||
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Progetto in Italia | Progetto Q1970834 in Italia |
Revision as of 22:10, 1 November 2020
Project Q1970834 in Italy
Language | Label | Description | Also known as |
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English | PROG HEAD — G1 — DOTT S3 — CRISPR/CAS9 AS A FUNCTIONAL ANALYSIS TECHNOLOGY — FN |
Project Q1970834 in Italy |
Statements
32,075.0 Euro
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64,150.0 Euro
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50.0 percent
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18 October 2017
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21 June 2021
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UNIVERSITA' DEGLI STUDI DI TRIESTE
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MESSA A PUNTO DI UN SISTEMA FUNZIONALE "IN VITRO" ATTRAVERSO LA TECNOLOGIA CRISPR/CAS9 PER STUDIARE L'EFFETTO DELLE VARIANTI GENICHE IDENTIFICATE IN MALATTIE MONOGENICHE DI CUI AD OGGI NON Ê STATO DESCRITTO UN MECCANISMO PATOGENETICO. QUESTA TECNOLOGIA RIVOLUZIONARIA PERMETTE DI INGEGNERIZZARE IL DNA DI INTERESSE IN TEMPI BREVI, CON ALTA EFFICIENZA E CON COSTI MODERATI, RISPETTO ALLE TECNOLOGIE DI INGEGNERIZZAZIONE BASATE SULLA RICOMBINAZIONE OMOLOGA COMUNEMENTE UTILIZZATE NEI LABORATORI DI GENETICA FINO A POCHI ANNI FA. HO SCELTO COME MODELLO DI PARTENZA LA AGAMMAGLOBULINEMIA X-LINKED, NOTA ANCHE COME SINDROME DI BRUTON, UNA RARA PATOLOGIA INFANTILE CHE COINVOLGE LA MATURAZIONE DEI LINFOCITI B E DI CONSEGUENZA UNA RIDOTTISSIMA PRODUZIONE DI IMMUNOGLOBULINE. QUESTA PATOLOGIA ESSENDO MONOGENICA CON CARATTERISTICHE DI EMIZIGOSITà , E COINVOLGENDO CELLULE EMATOPOIETICHE AMPIAMENTE UTILIZZATE E PROFONDAMENTE CONOSCIUTE, SI PRESENTA COME OTTIMO MODELLO PER LO STUDIO QUI PROPOSTO. (Italian)
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DEVELOPMENT OF AN ‘IN VITRO’ FUNCTIONAL SYSTEM USING CRISPR/CAS9 TO STUDY THE EFFECT OF THE GENE VARIANTS IDENTIFIED IN MONOGENICHE TO WHICH A PATHGENETIC MECHANISM HAS NOT BEEN DEVELOPED TO DATE. THIS REVOLUTIONARY TECHNOLOGY ALLOWS THE ENGINEERING OF DNA OF INTEREST AT SHORT NOTICE, WITH HIGH EFFICIENCY AND MODERATE COSTS, COMPARED TO ENGINEERING TECHNOLOGIES THAT ARE BASED ON RECOMBINATION COMMONLY USED IN GENETICS LABORATORIES UNTIL A FEW YEARS AGO. I HAVE CHOSEN AS THE STARTING POINT, THE AGAMMAGLOBULINEMIA X-LINKED, ALSO KNOWN AS BRUTON’S SYNDROME, A RARE CHILDHOOD MORBIDITY INVOLVING THE RIPENING OF THE LYMPHOCYTES B AND THEREFORE A VERY LOW PRODUCTION OF IMMUNOGLOBULINS. THIS PATHOLOGY, BEING MR MONGENICA WITH CHARACTERISTICS OF EMIOZIGIFIT, AND THE EXTENSIVE AND WIDELY KNOWN HAEMATOPOIETIC CELLS, IS A VERY GOOD MODEL FOR THE PROPOSED STUDY. (English)
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Identifiers
J93C17000120009
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