ãó äáéú

çãùåú åãéååçéí

çãùåú îäàøõ åîäòåìí

Novo Nordiski ôéúåç èéôåì âðèé çãù ìäîåôéìéä

ôåøñí áàúø çãùåú äîåôéìéä äéåí  áúàøéê 14.10.2019

https://hemophilianewstoday.com/2019/10/14/novo-nordisk-partners-bluebird-develop-gene-therapies-genetic-disorders

ä÷ãîä: ááéú äçåìéí úì äùåîø ðòøê ëéåí ðéñåé áøéôåé âðèé - ùäåà äçãøú âï ìúàé äëáã åìà ìúåê ä-DNA ùì äúàéí. ëê ùìàçø æîï ëåùø äééöåø éåøã ìàôñ åöøéê ùåá åùåá ìäçãéø àú äâï áàîöòåú äùìéç ùäåà åéøåñ. áîàîø ùìäìï îúåàø ôéúåç çãù ùì ùéëúåá âðèé ùðòùä áùéúåó ôòåìä áéï çáøú ðåáå ðåøãéñ÷ ìáìåáéøã áéå, ëê, ùòåøëéí îçãù àú ä-DNA åäúé÷åï àîåø ìäéåú ÷áåò åéöéá ìàåøê ëì çééå ùì äçåìä . *  *  *

ùéúåó ôòåìä áéï çáøú ðåáå ðåøãéñ÷ åáìåáéøã-áéå ìôéúåç èéôåì âðèé çãù ìäîåôéìéä åäôøòåú àçøåú îàú: â'åàðä ÷øáìéå MSc * ðåáå ðåøãéñ÷ äú÷ùøä áäñëí ùéúåó ôòåìä òí áìåáéøã-áéå ìôéúåç èéôåìéí çãùéí äîáåññéí òì òøéëú âðéí ìñãøú äôøòåú âðèéåú, ëåìì äîåôéìéä. äçáøåú ñéëîå ìòáåã éçã òì ôøåé÷è îç÷ø ùðîùê ùìåù ùðéí ùéúî÷ã áæéäåé åôéúåç ùì îåòîãéí ìèéôåì âðèé ìèéôåì áäîåôéìéä A , ñåâ ùì äîåôéìéä äðâøîú ëúåöàä îçåñø àå ú÷ìä ùì âåøí ä÷øéùä (ô÷èåø FVIII) áãí. àí éöìéç äôøåé÷è, äãáø òùåé ìäéåú äöòã äøàùåï áôéúåç èéôåì âðèé çãù ìäîåôéìéä A àùø éàôùø ìîèåôìéí ìçéåú ììà öåøê áäæø÷ä ùì ô÷èåøéí ìåøéã ëôé ùðòùä äéåí. ëãé ìäùéâ îèøä æå, äçå÷øéí éúî÷ãå áúçéìä áîöéàú ãøê ìú÷ï àú äéòãø äô÷èåø VIII, éòã äèéôåì äòé÷øé áäîåôéìéä A. éòãéí èéôåìééí àçøéí òùåééí ìäéáã÷ áùìá îàåçø éåúø. äôøåé÷è éùúîù áèëðåìåâééú îâä-èì ùááòìåú áìåáéøã-áéå. îâä-èì äí àðæéîéí äðéúðéí ìäúàîä àéùéú (çìáåðéí îéåçãéí) ùéù ìäí àú äéëåìú ìæäåú àæåøéí ñôöéôééí äîòðééðéí áâðåí (ëì äâðéí ä÷ééîéí á- DNA ùìðå) åìçúåê DNA. îñéáåú àìä äí ëìéí øáé òåöîä ùðéúï ìäùúîù áäí ëãé ìäåñéó, ìòøåê àå ìäôòéì âðéí ñôöéôééí äîòðééðéí àåúí. ôéìéô âøâåøé, äîãòï äøàùé ùì çáøú áìåáéøã-áéå , àîø áäåãòä ìòéúåï "áìåáéøã-áéå äú÷ãîä äú÷ãîåú àãéøä áàôùøåú ôìèôåøîú òøéëú âðéí in vivo äîáåññú òì èëðåìåâééú îâä-èì ùìðå, ëåìì äú÷ãîåú çùåáä áééöåø åèéäåø mRNA áàéëåú âáåää". "àðå îàîéðéí ëé ìèëðåìåâéä æå éù ôåèðöéàì ìéöåø âéùä îåáçðú îàåã ìèéôåì áîçìåú âðèéåú ÷ùåú øáåú," äåñéó. äùåúôåú úåñéó âí ìúé÷ ääîåôéìéä ùì ðåáå ðåøãéñ÷. îø÷åñ ùéðãìø, ñîðë"ì áëéø ìâéìåé úøåôåú òåìîé áðåáå ðåøãéñ÷, "àðå ùîçéí ìäëøéæ òì ùéúåó äôòåìä ùìðå òí áìåáéøã-áéå ùéëåìåúéä äîåëçåú áèéôåì âðèé éàôùøå ìãåø äáà ùì îåöøéí çãùðééí ìäùôéò îùîòåúéú òì çéé äçåìéí. àîø. "ùéúåó ôòåìä îç÷øé çùåá ùîèøúå ìäúîåãã òí îçìåú âðèéåú áøîú ä-DNA îù÷ó àú äîçåéáåú äîúîãú ùì ðåáå ðåøãéñ÷ ìäîöéà úøåôåú ìùéðåé îçìåú ùéëåìåú ìùðåú áàîú àú çééäí ùì àðùéí äçééí òí äîåôéìéä åîçìåú âðèéåú àçøåú," àîø. _____ * òì äëåúáú â'åàðä ÷øáìéå MSc â'åàðä îñééîú áéîéí àìä àú äãå÷èåøè ááéå-øôåàä åáîç÷ø ÷ìéðé áàåðéáøñéèú ãä ìéñáåàä. áðåñó äéà áòìú úåàø øàùåï ááéåìåâéä åúåàø ùðé ááéåìåâéä àáåìåöéåðéú åäúôúçåúéú îàåðéáøñéèú ãä ìéñáåàä. òáåãúä äúî÷ãä áäùôòä ùì àéúåú Wnt ìà-÷ðåðé áäúðäâåúí ä÷åì÷èéáéú ùì úàé äàðãåúì - úàéí äîøëéáéí àú øéøéú ëìé äãí - ùðîöàéí áçáì äèáåø ùì äéìåãéí. ä÷ãîä åôøùðåú: çééí åééñîï úøâåí îàðâìéú: àìé åéùðéöø äîàîø äî÷åøé áàðâìéú:

October 14, 2019 Novo Nordisk Partners with Bluebird Bio to Develop New Gene Therapies for Hemophilia, Other Disorders By Joana Carvalho, MSc. * Novo Nordisk has entered into a collaboration agreement with Bluebird Bio to develop new therapies based on gene editing for a series of genetic disorders, including hemophilia. The companies have agreed to work together on a three-year research project that will focus on the identification and development of gene therapy candidates to treat hemophilia A, a type of hemophilia caused by the lack or malfunction of clotting factor VIII (FVIII) in the blood. If successful, this project may be the first step in the development of a new gene therapy for hemophilia A which would allow patients to live without requiring constant factor replacement therapy. To achieve that goal, researchers will focus initially on finding a way to correct the lack of FVIII, the main therapy target for hemophilia A. Other therapeutic targets may be explored at a later stage. The project will use Bluebird Bio’s proprietary megaTAL technology. MegaTALs are customizable enzymes (special proteins) that have the ability to recognize specific regions of interest within the genome (all of the genes present in our DNA) and cut DNA. For these reasons, they are powerful tools that can be used to add, edit, or inactivate specific genes of interest. “Bluebird has made tremendous progress on enabling an in vivo gene editing platform based on our megaTAL technology, including important advances in high-quality mRNA production and purification,” Philip Gregory, chief scientific officer of Bluebird Bio, said in a news release. “We believe this technology has the potential to create a highly differentiated approach to the treatment of many severe genetic diseases,” he added. The partnership will also add to Novo Nordisk’s hemophilia portfolio. “We are pleased to announce our collaboration with Bluebird whose demonstrated capabilities in gene therapy will enable the next-generation of innovative products to make a significant impact on patients’ lives,” Marcus Schindler, senior vice president for global drug discovery at Novo Nordisk, said. “This important research collaboration aimed at addressing genetic diseases at the DNA level reflects Novo Nordisk’s enduring commitment and dedication to inventing disease-modifying medicines that can truly change the lives of people living with haemophilia and other genetic diseases,” he said. _____ * Joana Carvalho, MSc Joana is currently completing her PhD in Biomedicine and Clinical Research at Universidade de Lisboa. She also holds a BSc in Biology and an MSc in Evolutionary and Developmental Biology from Universidade de Lisboa. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that make up the lining of blood vessels — found in the umbilical cord of newborns.