{"id":1988,"date":"2018-06-19T12:29:12","date_gmt":"2018-06-19T17:29:12","guid":{"rendered":"https:\/\/restruct-lgmd-2021.pantheonsite.io\/?p=1988"},"modified":"2018-06-19T12:29:12","modified_gmt":"2018-06-19T17:29:12","slug":"lgmd-researcher-chamberlain","status":"publish","type":"post","link":"https:\/\/www.lgmd-info.org\/pt\/researcher-interview\/2018\/06\/19\/lgmd-researcher-chamberlain\/","title":{"rendered":"INVESTIGADOR DO LGMD: Jeffrey S. Chamberlain, Ph.D."},"content":{"rendered":"

\"LGMD EM DESTAQUE NA INVESTIGA\u00c7\u00c3O\"<\/strong><\/p>\n

Investigador LGMD:<\/strong> Jeffrey S. Chamberlain, Ph.D.\"\"<\/a><\/p>\n

Filia\u00e7\u00e3o:<\/strong>\u00a0 Faculdade de Medicina da Universidade de Washington<\/p>\n

Fun\u00e7\u00e3o ou cargo<\/strong>: Professor, C\u00e1tedra McCaw em Distrofia Muscular, Diretor, Centro de Investiga\u00e7\u00e3o em Distrofia Muscular Wellstone-Seattle<\/p>\n

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

Obtive uma licenciatura e um doutoramento em Bioqu\u00edmica e depois fiz um p\u00f3s-doutoramento em Gen\u00e9tica Molecular<\/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

Enquanto trabalhava no meu doutoramento, interessei-me pelo desenvolvimento muscular e comecei a aprender sobre doen\u00e7as musculares. Fiz um est\u00e1gio de p\u00f3s-doutoramento para estudar a distrofia muscular de Duchenne e fiquei interessado e intrigado com o conceito de terapia gen\u00e9tica. Quando comecei o meu pr\u00f3prio laborat\u00f3rio, decidi concentrar-me na terapia gen\u00e9tica para as distrofias musculares, bem como no estudo da fun\u00e7\u00e3o das prote\u00ednas defeituosas nas diferentes distrofias.<\/p>\n

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

Atualmente, estudo m\u00e9todos para a terapia gen\u00e9tica de doen\u00e7as musculares, incluindo vectores para entregar genes aos tecidos musculares, cassetes de regula\u00e7\u00e3o gen\u00e9tica para ligar e desligar genes em diferentes tecidos, e o papel do sistema imunit\u00e1rio nas distrofias e no contexto da terapia gen\u00e9tica. Estudo tamb\u00e9m a fun\u00e7\u00e3o e as intera\u00e7\u00f5es de diferentes prote\u00ednas que contribuem para a fun\u00e7\u00e3o e a doen\u00e7a muscular, como a distrofina e a FKRP.<\/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 as LGMD ou para os m\u00e9dicos em geral?<\/strong><\/p>\n

O nosso trabalho \u00e9 simultaneamente cient\u00edfico e centrado no tratamento. Acreditamos que a melhor forma de desenvolver tratamentos eficazes \u00e9 ter uma compreens\u00e3o pormenorizada da biologia b\u00e1sica subjacente a v\u00e1rias doen\u00e7as musculares. Utilizamos esta investiga\u00e7\u00e3o b\u00e1sica para orientar o desenvolvimento de genes e ve\u00edculos de entrega necess\u00e1rios para uma terapia gen\u00e9tica segura e eficaz.<\/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

A terapia g\u00e9nica deixou de ser um conceito futurista para se tornar uma abordagem promissora no tratamento de doen\u00e7as heredit\u00e1rias. Atualmente, v\u00e1rias terapias gen\u00e9ticas foram aprovadas para uso comercial e podem ser prescritas por m\u00e9dicos. Penso que as terapias gen\u00e9ticas s\u00e3o a abordagem mais promissora para tratar a maioria dos diferentes tipos de distrofia muscular. Tamb\u00e9m \u00e9 importante perceber que os desafios que limitam o desenvolvimento da terapia gen\u00e9tica para muitas distrofias s\u00e3o os mesmos para as v\u00e1rias doen\u00e7as, e que o progresso numa delas ajuda a aproximar os tratamentos da cl\u00ednica para todas as distrofias. Est\u00e3o em curso ensaios de terapia g\u00e9nica para v\u00e1rias distrofias musculares e muitas outras est\u00e3o prestes a ser testadas. Estamos \u00e0 beira de uma revolu\u00e7\u00e3o no desenvolvimento e aplica\u00e7\u00e3o de novas terapias gen\u00e9ticas e penso que esta \u00e9 uma altura fant\u00e1stica para ver estas novas tecnologias come\u00e7arem finalmente a ajudar os doentes e as suas fam\u00edlias.<\/p>\n

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

H\u00e1 30 anos que trabalho com a distrofia muscular e estou finalmente a assistir \u00e0 aplica\u00e7\u00e3o e ao funcionamento de novas terapias na cl\u00ednica. Esta \u00e9 a altura mais excitante da minha carreira e estou entusiasmado por poder contribuir para o desenvolvimento de novas abordagens para o tratamento de diferentes tipos de doen\u00e7as musculares.<\/p>\n

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

O financiamento da investiga\u00e7\u00e3o cient\u00edfica est\u00e1 a diminuir em todo o pa\u00eds e, em muitos quadrantes, parece haver quase uma guerra contra as ideias e os princ\u00edpios cient\u00edficos que s\u00e3o ensinados e explorados. A coisa mais importante que os doentes e os familiares podem fazer \u00e9 encorajar os membros dos seus governos locais e nacionais a apoiar a investiga\u00e7\u00e3o cient\u00edfica nas universidades de todo o pa\u00eds. Quase todas as abordagens inovadoras e novas \u00e0s terapias, tais como as terapias gen\u00e9ticas e celulares, est\u00e3o a ser desenvolvidas exclusivamente nas Universidades antes de serem eventualmente adoptadas pela biotecnologia e pelas grandes empresas farmac\u00eauticas.<\/p>\n

 <\/p>\n

* Por favor, GOSTEI, COMENTEI e PARTILHEI esta publica\u00e7\u00e3o para ajudar a aumentar a sensibiliza\u00e7\u00e3o para a LGMD!<\/p>\n

* Para saber mais sobre a Distrofia Muscular das Cinturas dos Membros (LGMD) ou para ler mais \"Entrevistas em Destaque\", visite o nosso s\u00edtio Web em https:\/\/lgmd-info.org<\/a><\/p>\n

#CureLGMD<\/p>\n

#LgmdDay2018<\/p>","protected":false},"excerpt":{"rendered":"

“LGMD SPOTLIGHT ON RESEARCH” LGMD Researcher: Jeffrey S. Chamberlain, Ph.D. […]<\/p>","protected":false},"author":1,"featured_media":1987,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[91,92],"tags":[],"class_list":["post-1988","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-researcher-interview","category-researcher"],"acf":[],"yoast_head":"\nLGMD RESEARCHER: Jeffrey S. 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