{"id":5832,"date":"2022-07-06T03:18:54","date_gmt":"2022-07-06T03:18:54","guid":{"rendered":"https:\/\/www.lgmd-info.org\/?p=5832"},"modified":"2024-02-09T14:23:53","modified_gmt":"2024-02-09T14:23:53","slug":"lgmd-researcher-pia-elustondo","status":"publish","type":"post","link":"https:\/\/www.lgmd-info.org\/pt\/researcher-interview\/2022\/07\/06\/lgmd-researcher-pia-elustondo\/","title":{"rendered":"INVESTIGADORA LGMD: Pia Elustondo"},"content":{"rendered":"

\"Pia<\/p>\n

INVESTIGADOR DA LGMD:\u00a0 <\/strong>\u00a0Dra. Pia Elustondo<\/p>\n

Filia\u00e7\u00e3o:\u00a0 <\/strong>AGADA Bioci\u00eancias<\/p>\n

Fun\u00e7\u00e3o ou cargo:\u00a0 <\/strong>Diretor de Investiga\u00e7\u00e3o<\/p>\n

Que educa\u00e7\u00e3o e forma\u00e7\u00e3o teve para chegar \u00e0 sua posi\u00e7\u00e3o atual?<\/strong><\/p>\n

Tenho uma vasta forma\u00e7\u00e3o; estudei bioqu\u00edmica e mecanismos de doen\u00e7a humana durante a minha licenciatura e p\u00f3s-gradua\u00e7\u00e3o. Durante os meus estudos de p\u00f3s-doutoramento, concentrei-me na fun\u00e7\u00e3o mitocondrial em modelos de AVC e doen\u00e7as neurodegenerativas (Niemann Pick) antes de me juntar \u00e0 comunidade da distrofia muscular. Queria expandir a minha experi\u00eancia para outras doen\u00e7as, como as distrofias musculares, e, em 2018, juntei-me \u00e0 AGADA Biosciences, uma empresa especializada em distrofias neuromusculares. Comecei numa posi\u00e7\u00e3o de cientista e rapidamente cresci para a posi\u00e7\u00e3o de Diretor de Investiga\u00e7\u00e3o. Na minha fun\u00e7\u00e3o, supervisiono investigadores que trabalham em modelos de distrofia muscular em ratos e peixes-zebra.<\/p>\n

O que o levou a seguir uma carreira de investiga\u00e7\u00e3o e a estudar a distrofia muscular em particular?<\/strong><\/p>\n

A minha curiosidade levou-me a fazer investiga\u00e7\u00e3o e o meu interesse pela biologia das mitoc\u00f4ndrias ligou-me \u00e0 distrofia muscular, porque as mitoc\u00f4ndrias desempenham um papel importante na fun\u00e7\u00e3o muscular e, se n\u00e3o funcionarem bem, podem provocar fraqueza muscular e fadiga. A AGADA Biosciences foi criada para facilitar especificamente o desenvolvimento de medicamentos para doen\u00e7as musculares raras, incluindo a LGMD. Um dos objectivos da AGADA \u00e9 formar e desenvolver uma for\u00e7a de trabalho cient\u00edfica que se concentre na distrofia muscular. Seguir uma carreira de investiga\u00e7\u00e3o na \u00e1rea da distrofia muscular e formar cientistas nesta \u00e1rea cumprem os meus objectivos profissionais.<\/p>\n

Que temas est\u00e3o a estudar?<\/strong><\/p>\n

Investigamos as raz\u00f5es que levam \u00e0 disfun\u00e7\u00e3o muscular nas distrofias musculares. Geramos e caracterizamos sistematicamente a progress\u00e3o da doen\u00e7a e a efic\u00e1cia dos medicamentos em modelos animais, incluindo ratinhos e peixe-zebra. O nosso objetivo \u00e9 analisar e identificar interven\u00e7\u00f5es (medicamentos, terapia gen\u00e9tica, produtos biol\u00f3gicos, etc.) que melhorem potencialmente a fun\u00e7\u00e3o muscular nestas doen\u00e7as. Al\u00e9m disso, estudamos tamb\u00e9m os resultados bioqu\u00edmicos para avaliar a efic\u00e1cia dos medicamentos em doentes que participam em ensaios cl\u00ednicos de distrofia muscular.<\/p>\n

Como \u00e9 que o seu trabalho vai ajudar os doentes? \u00c9 de natureza mais cient\u00edfica ou poder\u00e1 tornar-se um tratamento para a LGMD?<\/strong>s ou MD<\/strong>s em geral?<\/strong><\/p>\n

O nosso objetivo \u00e9 encontrar interven\u00e7\u00f5es seguras e eficazes em modelos animais de distrofias musculares. Queremos fornecer dados que nos d\u00eaem confian\u00e7a para fazer a transi\u00e7\u00e3o segura de um medicamento para ensaios cl\u00ednicos em humanos. Alguns dos nossos estudos s\u00e3o de natureza cient\u00edfica, mas o nosso objetivo final \u00e9 utilizar estes resultados como biomarcadores da progress\u00e3o da doen\u00e7a e da resposta \u00e0 terap\u00eautica.<\/p>\n

O que gostaria que os doentes e outras pessoas interessadas na LGMD soubessem sobre a investiga\u00e7\u00e3o (os seus pr\u00f3prios projectos e a \u00e1rea em geral)?<\/strong><\/p>\n

Queremos que os doentes saibam que a comunidade cient\u00edfica fez progressos significativos na descoberta de tratamentos para distrofias musculares como a DMD. Isto d\u00e1-nos confian\u00e7a de que \u00e9 poss\u00edvel fazer progressos semelhantes na descoberta de tratamentos eficazes para as LGMD. A nossa investiga\u00e7\u00e3o utiliza as mais recentes tecnologias, como a CRISPR, para criar modelos de peixe-zebra para as LGMD, que permitir\u00e3o testar medicamentos de forma r\u00e1pida e econ\u00f3mica.<\/p>\n

O que o inspira a continuar a trabalhar neste dom\u00ednio? <\/strong><\/p>\n

Quero que a qualidade de vida dos doentes e dos prestadores de cuidados melhore; \u00e9 isso que me inspira a continuar a fazer investiga\u00e7\u00e3o.<\/p>\n

Como \u00e9 que os doentes o podem encorajar e ajudar no seu trabalho?<\/strong><\/p>\n

Os doentes apoiam estas iniciativas atrav\u00e9s da participa\u00e7\u00e3o em ensaios cl\u00ednicos, da sensibiliza\u00e7\u00e3o e da angaria\u00e7\u00e3o de fundos. Isto \u00e9 muito motivador para mim e, diria eu, para toda a comunidade de investiga\u00e7\u00e3o.<\/p>\n<\/div><\/div><\/div><\/div><\/div>","protected":false},"excerpt":{"rendered":"","protected":false},"author":3,"featured_media":5831,"comment_status":"closed","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[91,92],"tags":[],"class_list":["post-5832","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-researcher-interview","category-researcher"],"acf":[],"yoast_head":"\nLGMD RESEARCHER: Pia Elustondo - LGMD Awareness Foundation<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/www.lgmd-info.org\/pt\/researcher-interview\/2022\/07\/06\/lgmd-researcher-pia-elustondo\/\" \/>\n<meta property=\"og:locale\" content=\"pt_PT\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"LGMD RESEARCHER: Pia Elustondo - 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