Científicos israelíes desarrollaron una nueva terapia para la sordera. Reemplazar genes defectuosos que causan una pérdida auditiva gradual tuvo éxito en ratones y podría conducir al tratamiento de bebés con mutaciones similares.
Suministrar material genético sano a las células del oído interno de ratones con un defecto genético que causa sordera permite que las células funcionen normalmente informó Israel21c.
Así lo indicó un nuevo estudio de la Universidad de Tel Aviv (TAU). Reveló que el nuevo tratamiento evitó el deterioro gradual de la audición en estos ratones. Esto es algo que podría dar lugar a un gran avance en el tratamiento de niños que nacen con diversas mutaciones que eventualmente causan hipoacusia.
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La investigación fue publicada el 22 de diciembre. La dirigió la profesora Karen Avraham del Departamento de Genética Molecular Humana y Bioquímica de la Facultad de Medicina y Neurociencia Sackler de TAU.
La sordera es la discapacidad sensorial más común en todo el mundo. De acuerdo con la Organización Mundial de la Salud, cerca de 500 millones de personas tienen pérdida auditiva. Sse espera que esta cifra se duplique en las próximas décadas.
Las estadísticas también indican que uno de cada 200 niños nace con una discapacidad auditiva y uno de cada 1.000 nace totalmente sordo.
En aproximadamente la mitad de estos casos, una mutación genética causa la hipoacusia, y de hecho 100 genes diferentes están asociados con la sordera hereditaria.
“En el estudio nos enfocamos en la hipoacusia genética causada por una mutación en el gen SYNE4. Es una sordera poco común descubierta por nuestro laboratorio hace varios años en dos familias israelíes, explicó Avraham.
«Desde entonces identificada en Turquía y también en el Reino Unido también. Los niños que heredan el gen defectuoso de ambos padres nacen con una audición normal pero gradualmente pierden la audición durante la niñez, agregó.
«La mutación provoca una ubicación incorrecta de los núcleos celulares en las células ciliadas dentro de la cóclea del oído interno. Los mismos sirven como receptores de ondas sonoras y son esenciales para la audición. Este defecto conduce a la degeneración y eventual muerte de las células ciliadas”, dijo.
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Una acción reparadora
El laboratorio de Avraham uso una tecnología innovadora de terapia génica. Allí crearon un virus sintético inofensivo que sirvió como “vehículo de entrega” de una versión normal del gen defectuoso. Tanto en el modelo de ratón como en las familias humanas afectadas.
El tratamiento se administró poco después del nacimiento de los ratones y luego se controló la audición mediante pruebas fisiológicas y de comportamiento.
A su vez, el profesor Jeffrey Holt del hospital de niños de Boston y la Escuela de Medicina de Harvard colaboró en el estudio. Manifestó que los descubrimientos son muy prometedores. Dijo que los ratones tratados “desarrollaron una audición normal, con una sensibilidad casi idéntica a la de los ratones sanos que no tienen la mutación”.
Ahora, los científicos ya desarrollan terapias similares para otras mutaciones que causan sordera.
“Este es un estudio importante», indicó el doctor Wade Chien del Instituto Nacional de la Sordera y Otros Trastornos de la Comunicación de EEUU. «Muestra que la terapia génica del oído interno se puede aplicar de manera efectiva. Como por ejemplo a un modelo de ratón de sordera SYNE4 para rescatar la audición”, agregó.
Chien afirmó: “La magnitud de la recuperación auditiva es impresionante. Este estudio es parte de un creciente cuerpo de estudio que muestra que la terapia génica se puede aplicar con éxito. En este caso a modelos de ratón de pérdida auditiva hereditaria. Ilustra el enorme potencial de la terapia génica como tratamiento para la sordera”.
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El estudio contó con diversos apoyos. La Fundación Binacional de Ciencias Estados Unidos-Israel, El Instituto Nacional de Salud y el Consejo Europeo de Investigación. También el Programa de Asociación de Medicina de Precisión de Israel de la Fundación de Ciencias de Israel.
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Эта публикация погружает вас в мир увлекательных фактов и удивительных открытий. Мы расскажем о ключевых событиях, которые изменили ход истории, и приоткроем завесу над научными достижениями, которые вдохновили миллионы. Узнайте, чему может научить нас прошлое и как применить эти знания в будущем.
Подробнее тут – https://vivod-iz-zapoya-2.ru/