Argumentos para investigar en ciencia El paradigmático ... · SCIENTISTS have identified hundreds...
Transcript of Argumentos para investigar en ciencia El paradigmático ... · SCIENTISTS have identified hundreds...
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Francisco J. M. Mojica
Argumentos para investigar en cienciaEl paradigmático caso de los sistemas CRISPR
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De This vector version: Eric Gaba (Sting - fr:Sting) - NASA Astrobiology Institute, found in an article(Source unavailable), Dominio público, https://commons.wikimedia.org/w/index.php?curid=26075698
Procariotas Eucariotas
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Genoma (ADN)
CRISPR CRISPR(Clustered Regularly Interspaced Short Palindromic Repeats)
Espaciador
Espaciadores
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1992: 1os Estudios
Salinas Solares. Santa Pola (Alicante)
Haloferax spp.
Boán, I.F. Universidad de Alicante
Francisco E.Rodríguez-Valera
GuadalupeJuez-Pérez
Martin Nieto, J. Universidad de Alicante
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GTTACAGACGAACCCTAGTTGGGTTGAAGCGAACAGGATGGCGAACCGGTGTCTGCACCAGTTGTTACAGACGAACCCTAGTTGGGTTGAAGCCACGACAATCAAGTCTGGTTGCATGGCGACACGGAGTTACAGACGAACCCTAGTTGGGTTGAAGCCTGTGCCTCCAGCGGCCGTCAGACAGTCGCATCCGAGTTACAGACGAACCCTAGTTGGGTTGAAGCAAGAAGCCGCTCGCCGTCCTCGATGACGGGCGGGCGGTTACAGACGAACCCTAGTTGGGTTGAAGCGACAAGACTCGCGACGAAGCCGAGTCGAAACGCCGCGTTACAGACGAACCCTAGTTGGGTTGAAGCCTCTTTATCCCTCCTGCCCGAATGTCTACGAATATCGTTACAGACGAACCCTAGTTGGGTTGAAGCGAACCCACTGGTGAAGAAAAAGTTGTAGAGACCCTAGTTACAGACGAATCCCTAGTTGGGTTGAAGCACGACAATCAAGTCTGGTTACATGGCGACAGGATGGGTTACAGACGAACCCTAGTTGGGTTGAAGCTTCCACAACGTCGGGGAGGGCGAAATTAGCCAAGCAGTTACAGACGAACCCTAGTTGGGTTGAAGC
1992: 1os Estudios
Mojica. FJM. Tesis Doctoral (1993)
A C G T
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¿Qué función desempeñan?
Mojica et al. Molecular Microbiology (1995)
Mojica, FJM.; Tesis Doctoral (1993)
ARN
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¿
¿
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2000: Una nueva familia de repeticiones
Francisco J.M. Mojica et al. Molecular Microbiology (2000)
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ClusteredRegularlyInterspacedShortPalindromicRepeats
CRISPR-Cas systems, Barrangou and van der Oost (Springer-Verlag Berlin Heidelberg 2013) With permission of Springer.
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Proteínas Cas
C R I S P R
Sistemas CRISPR
Genes cas (CRISPR associated )
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Proteínas Cas
C R I S P R
Sistemas CRISPR
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Sistemas CRISPR
Proteínas Cas
C R I S P R
Sistema de Inmunidad Adquirida
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Mojica & Almendros. Investigación y Ciencia. 2017
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Infección
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Inmunización
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Célula Inmunizada
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Producción de guías
ARN guía
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Reinfección
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Reconocimiento
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Corte
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Inactivación
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Aplicaciones de CRISPR en procariotas
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Vacunación
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Almacenamiento de datos
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IIIIIIIIIIII
Cas9
ARN guía
IIIIIIIIIIIIIIIIIIIIIIIIIIIIIIIIIIIIIIADN
La Tecnología CRISPR
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Cas9
ARN guía
IIIIIIIIIIIIIIIIIIIIIIIIIIIIIIIIIIIIIIADNIIIIIIIIIIII
La Tecnología CRISPR
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Matar bacterias
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Aplicaciones de la tecnología CRISPR en cualquier ser vivo
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Cas9
ARN guía
dCas9
• Diagnóstico molecular
• Control de la actividad genética
• Reescribir el ARN
• Actuar sobre el ADN
Visualización
Arquitectura
Emplazamiento
Cambios epigenéticos
Edición genética
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Cas9
ARN guía
dCas9
• Diagnóstico molecular
• Control de la actividad genética
• Reescribir el ARN
• Actuar sobre el ADN
Visualización
Arquitectura
Emplazamiento
Cambios epigenéticos
Edición genética
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Diagnóstico Molecular
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Edición Genética
-- ACTGGGGGCCCATGGTACCGGTTAATTACCGGTAC --(Esta proteína se encarga de sintetizar colesterol)
-- ACTGGGGGCCCTACCATGAGGTTAATTACCGGTAC --(Esta proteína ya no puede sintetizar colesterol)
-- ACTGGGGGCCCATGGTACCGGTTAATT--------GTAC--(Esta proteína no es funcional)
-- ACTGGGGGCCCATGGTACCGGTTAATTACCGGTACCATACCG --(Esta proteína se encarga de sintetizar diversos lípidos)
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• Biotecnología
• Productividad y calidad de alimentos
• Salud y bienestar animal
• Salud humana
• Prevención y tratamiento de infecciones
• Alteraciones genéticas
• Diagnóstico de enfermedades
Aplicacionesde la edición
genética en …
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Agricultura• Frutas sin semillas
• Cultivos con mayor productividad
• Composición modificada (sin gluten, contenido oleico…)
• Inmunes a enfermedades …
• Cultivos resistentes
• ….
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Ganadería
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Estudio y prevención de enfermedades
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Estudio y prevención de enfermedades
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Estudio y prevención de enfermedades
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CRISPR como agente terapéutico en animales
• Deficiencia de ornitina transcarbamilasa• Enfermedad granulomatosa crónica• Distrofia muscular de Duchenne• Amaurosis congénita de Leber• Enfermedad de Huntington• Tirosinemia hereditaria• Retinosis pigmentaria• Cataratas congénitas• Hemofilias• Diabetes• Cáncer• VIH• …
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Ensayos clínicos con CRISPR en humanos
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Mojica & Almendros. Investigación y Ciencia. 2017