{"id":83872,"date":"2021-04-30T09:42:32","date_gmt":"2021-04-30T07:42:32","guid":{"rendered":"https:\/\/ibecbarcelona.eu\/?p=83872"},"modified":"2021-04-30T10:29:57","modified_gmt":"2021-04-30T08:29:57","slug":"cap-a-un-tractament-per-a-la-distrofia-miotonica-desenvolupen-el-primer-model-3d-amb-cel%c2%b7lules-de-pacients","status":"publish","type":"post","link":"https:\/\/ibecbarcelona.eu\/ca\/cap-a-un-tractament-per-a-la-distrofia-miotonica-desenvolupen-el-primer-model-3d-amb-cel%c2%b7lules-de-pacients\/","title":{"rendered":"Cap a un tractament per a la distr\u00f2fia miot\u00f2nica: primer model 3D amb c\u00e8l\u00b7lules de pacients"},"content":{"rendered":"

Aquest avan\u00e7 permetr\u00e0 dissenyar tractaments personalitzats i m\u00e9s efectius, i testar futurs f\u00e0rmacs d’una manera molt m\u00e9s eficient.<\/span><\/span><\/p>\n

\"\"
Investigadors de l’IBEC Juanma Fern\u00e1ndez, Javier Ram\u00f3n i Xiomara Fern\u00e1ndez<\/figcaption><\/figure>\n

La distr\u00f2fia <\/span>miot\u00f2nica<\/span> \u00e9s una malaltia neuromuscular degenerativa heredit\u00e0ria que es manifesta principalment en adults, afectant unes 50.000 persones a Espanya. Els s\u00edmptomes varien des <\/span>de <\/span>dificultat per caminar fins a greus problemes neurol\u00f2gics, i condueixen a una incapacitaci\u00f3 progressiva que per desgr\u00e0cia porta a la cadira de rodes a molts dels afectats. Aquesta malaltia \u00e9s molt heterog\u00e8nia entre pacients (edat d’inici, progressi\u00f3, transmissi\u00f3 heredit\u00e0ria, m\u00fasculs afectats) el que fa especialment complex el desenvolupament de tractaments gen\u00e8rics.<\/span>\u00a0<\/span><\/p>\n

Actualment, els f\u00e0rmacs contra la distr\u00f2fia <\/span>miot\u00f2nica<\/span> es desenvolupen en models animals com la <\/span>Drosophila<\/span>, la mosca de<\/span>l<\/span> vinagre, o en cultius cel\u00b7lulars en 2D. M\u00e9s tard es proven en ratolins i, finalment en pacients. Utilitzant aquests enfocaments s’han desenvolupat alguns f\u00e0rmacs que desafortunadament no han pogut arribar a les \u00faltimes fases d’assajos cl\u00ednics perqu\u00e8 no eren efectius en pacients, posant de manifest la dificultat d’extrapolar resultats obtinguts amb animals a \u00e9ssers humans.<\/span>\u00a0<\/span><\/p>\n

Ara, l’equip liderat pel Professor d’Investiga<\/span>ci\u00f3<\/span> ICREA i cap del grup <\/span>Biosensors per Bioenginyeria<\/span><\/a> a l’IBEC, Javier Ram\u00f3n, i per l’investigador <\/span>postdoctoral<\/span> del seu equip Juan M. Fern\u00e1ndez, <\/span>ha desenvolupat per primera vegada un model tridimensional per a la distr\u00f2fia miot\u00f2nica, utilitzant t\u00e8cniques de bioenginyeria i c\u00e8l\u00b7lules de pacient<\/strong>.<\/strong> El treball, que forma part de la tesi doctoral de la investigadora <\/span>Xiomara<\/span> Fern\u00e1ndez de l’IBEC<\/span> i<\/span> s’ha realitzat en col\u00b7laboraci\u00f3 amb investigadors de l’institut de recerca sanit\u00e0ria INCLIVA de Val\u00e8ncia<\/span>,<\/span> s’ha publicat recentment a la revista <\/span>Biofabrication<\/span><\/i><\/a> i <\/span>obre les portes a trobar tractaments personalitzats i efectius contra aquesta malaltia rara i fins a<\/span>ra <\/span>sense cura. Aquest treball s’ha finan\u00e7at en part amb fons d’una <\/span>campanya de recaptaci\u00f3 de fons<\/span><\/a> llan\u00e7ada per l’IBEC des de finals de 2017 fins a finals de l’2019.<\/span>\u00a0<\/span><\/p>\n

El nou model 3D per a la distr\u00f2fia miot\u00f2nica\u00a0<\/strong><\/h2>\n

\"\"<\/span><\/p>\n

Els investigadors han partit de fibroblasts de la pell d’un pacient, c\u00e8l\u00b7lules del teixit connectiu encarregat de mantenir<\/span> la seva <\/span>estructura, <\/span>que<\/span> van ser pr\u00e8viament “<\/span>reprogramats<\/span>” perqu\u00e8 es convertissin en mioblasts, c\u00e8l\u00b7lules precursores de<\/span>l <\/span>m\u00fascul. Aquests mioblasts es van posar dins d’una matriu de gelatina i cel\u00b7lulosa i la barreja es va sotmetre a una mena de segell de goma per donar-li una forma de rectangles allargats, simulant les fibres musculars. Posteriorment la matriu es va incubar sota les condicions ideals perqu\u00e8 els mioblasts seguissin el seu desenvolupament.<\/span>\u00a0<\/span><\/p>\n

 <\/p>\n

Imatge: <\/strong><\/span>Imatge<\/span> de <\/span>microsc\u00f2pia<\/span> confocal<\/span> de <\/span>m\u00fascul<\/span> in vitro <\/span>desenvolupat<\/span> a partir de <\/span>c\u00e8l\u00b7lules<\/span> de <\/span>pacient<\/span> en <\/span>qu\u00e8<\/span> s’aprecia<\/span> la <\/span>tridimensionalitat<\/span> de la roba. En <\/span>blau<\/span> es marquen <\/span>els<\/span> nuclis<\/span> cel\u00b7lulars<\/span> i en <\/span>vermell<\/span> el <\/span>m\u00fascul<\/span> esquel\u00e8tic<\/span>.<\/span>\u00a0<\/span><\/h6>\n
\n

E<\/span><\/span>l que observem \u00e9s que, a difer\u00e8ncia dels cultius cel\u00b7lulars cl\u00e0ssics, les c\u00e8l\u00b7lules de pacient en el nostre model s\u00f3n capa<\/span><\/span>ce<\/span><\/span>s de fusionar-<\/span><\/span>se <\/span><\/span>formant fibres musculars que adopten la forma 3D <\/span><\/span>observada<\/span><\/span> en els m\u00fasculs i essencial per al seu funcionament<\/span><\/span>. <\/strong>
\n\u00a0<\/strong><\/p>\n

Juan M. Fernandez <\/strong>
\n<\/span><\/p>\n<\/blockquote>\n

Possibilitat de provar f\u00e0rmacs de forma personalitzada i r\u00e0pida\u00a0<\/strong><\/h2>\n

Per avaluar la funcionalitat del nou model 3D, els investigadors han aplicat a les fibres musculars un f\u00e0rmac, AntagomiR-23b, pr\u00e8viament desenvolupat per alguns autors d’aquest treball, i que en un futur no molt lluny\u00e0 entrar\u00e0 en fase d’assajos cl\u00ednics a trav\u00e9s de l’empresa <\/span>Arthex<\/span> Biotech<\/span><\/a> que continua el progr\u00e9s de la ter\u00e0pia. Van observar que tant les caracter\u00edstiques moleculars com estructurals de les fibres derivades de<\/span>l <\/span>pacient van recuperar els seus par\u00e0metres normals <\/span>despr\u00e8s del <\/span>tractament<\/span>, validant la seva utilitzaci\u00f3 per al desenvolupament de nous f\u00e0rmacs.<\/span>\u00a0<\/span><\/p>\n

A m\u00e9s, aquest model t\u00e9 un cost redu\u00eft, la seva implementaci\u00f3 \u00e9s simple i pot accelerar el proc\u00e9s de desenvolupament de nous tractaments, ja que \u00e9s m\u00e9s r\u00e0pid comparat amb l’\u00fas d’animals. Representa un gran pas cap a la medicina personalitzada, ja que tenir en compte l’enorme variabilitat entre pacients \u00e9s clau en el moment de buscar tractaments per aquesta malaltia.<\/span>\u00a0<\/span><\/p>\n

Aquest nou model permetr\u00e0 provar diferents f\u00e0rmacs i administrar al pacient el que ha donat millor resultat amb les seves pr\u00f2pies c\u00e8l\u00b7lules. D’aquesta manera el tractament ser\u00e0 personalitzat, m\u00e9s efectiu i <\/span><\/span>a <\/span><\/span>m\u00e9s es prescindeix de l’\u00fas d’animals<\/span><\/span>.\u00a0<\/strong>
\n\u00a0<\/strong><\/p>\n

Xiomara Fernandez\u00a0<\/strong>
\n<\/span><\/p>\n<\/blockquote>\n

La distr\u00f2fia miot\u00f2nica\u00a0<\/strong><\/h2>\n

La malaltia <\/span>\u00e9s<\/span> causada per una mutaci\u00f3 en el gen DMPK (<\/span>Dystrophia<\/span> Myotonica<\/span> Protein<\/span> Kinase<\/span>) que causa el “segrest” de prote\u00efnes essencials per al correcte funcionament dels m\u00fasculs, especialment les prote\u00efnes “<\/span>Muscleblind<\/span>“. A<\/span>l<\/span> reduir la seva disponibilitat no pot realitzar normalment les seves funcions, entre elles fer la transici\u00f3 d’altres prote\u00efnes d’una fase fetal o infantil a una fase adulta. La conseq\u00fc\u00e8ncia \u00e9s que els pacients tenen prote\u00efnes que no funcionen com les d’un adult. Dos exemples s\u00f3n el receptor d’insulina, que en pacients de distr\u00f2fia <\/span>miot\u00f2nica<\/span> no funciona correctament i causa diabetis, i la prote\u00efna que regula l’entrada i sortida de clor de les c\u00e8l\u00b7lules musculars, els coneguts com a canals de clor. El seu mal funcionament \u00e9s la causa <\/span>de <\/span>la miotonia, el s\u00edmptoma que defineix la malaltia i que fa que els pacients puguin contraure el m\u00fascul, per\u00f2 tinguin molta dificultat per relaxar-lo.<\/span>\u00a0<\/span><\/p>\n

\n
\n

Article de refer\u00e8ncia: <\/strong>Xiomara Fern\u00e1ndez-Garibay, Mar\u00eda A. Ortega, Estefan\u00eda Cerro-Herreros, Jordi Comelles, Elena Mart\u00ednez, Rub\u00e9n Artero, Juan M. Fern\u00e1ndez-Costa and Javier Ramon. Bioengineered in vitro 3D model of myotonic dystrophy type 1 human skeletal muscle.<\/a><\/u> <\/span>Biofabrication (2021), 13 (3), 035035.<\/span><\/p>\n

\u00a0<\/span><\/p>\n","protected":false},"excerpt":{"rendered":"

Investigadors de l’IBEC liderats per Javier Ram\u00f3n i Juan M. Fern\u00e1ndez desenvolupen el primer model tridimensional per a la distr\u00f2fia miot\u00f2nica, una malaltia rara i sense cura. El model combina c\u00e8l\u00b7lules de pacient i t\u00e8cniques de bioenginyeria i representa un gran aven\u00e7 respecte a la utilitzaci\u00f3 d’animals i de cultius cel\u00b7lulars.<\/h3>\n","protected":false},"author":4,"featured_media":83863,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[96],"tags":[1131],"post_formats":[],"class_list":["post-83872","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-noticies-de-recerca","tag-javier-ramon-ca"],"yoast_head":"\nCap a un tractament per a la distr\u00f2fia miot\u00f2nica: primer model 3D amb c\u00e8l\u00b7lules de pacients - Institute for Bioengineering of Catalonia<\/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:\/\/ibecbarcelona.eu\/ca\/cap-a-un-tractament-per-a-la-distrofia-miotonica-desenvolupen-el-primer-model-3d-amb-cel\u00b7lules-de-pacients\/\" \/>\n<meta property=\"og:locale\" content=\"ca_ES\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Cap a un tractament per a la distr\u00f2fia miot\u00f2nica: primer model 3D amb c\u00e8l\u00b7lules de pacients - Institute for Bioengineering of Catalonia\" \/>\n<meta property=\"og:description\" content=\"Investigadors de l'IBEC liderats per Javier Ram\u00f3n i Juan M. 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