Gene Therapy for Myotubular Myopathy: Early Signs of Success! | DNA Science Blog

Parents cherish developmental milestones, from a newborn’s grip of an offered finger; to an infant’s holding her head up the first time; to rolling over, creeping, and crawling; then to standing, cruising, and finally walking. Even kicking during a diaper change or yowling requires muscle strength and coordination. But a boy with X-linked myotubular myopathy (MTM) is so weak that even breathing is a huge struggle. If a baby survives the initial hospital stay, care at home becomes a full-time job and is only supportive, delaying the inevitable. That grim picture may be changing. Thanks to the efforts of parents, geneticists, physicians, and veterinarians, a gene therapy for MTM has had early encouraging results as a few treated boys are maxing out on standard tests of neuromuscular function (CHOP INTEND) and breathing (maximal inspiratory pressure or MIP). One boy who’d only received nutrition through a stomach tube is now feeding himself; another can pick up and throw a ball. The story

Source: Gene Therapy for Myotubular Myopathy: Early Signs of Success! | DNA Science Blog

NVGCT members participate in IOVC 2018

Scientists from the Netherlands participated in the 11th International Oncolytic Virus Conference in Oxford, UK (April 9-12, 2018). This short movie gives an impression of the meeting, with special attention for the contributions by delegates from the Netherlands and their reflections on the meeting.


As you may know, talks by invited speakers and selected proffered papers were filmed and many of them are now available through “My Congress Materials” in your profile on the IOVC website. To watch the talks, please log in to your profile, My Congress Materials will show up in red on the right hand side. Click this, then view the 11th International Oncolytic Virus Conference and go to Other Materials, where you will find a link to the video.


If you are aware of anyone in your institution who was unable to come to IOVC 2018, but would like to watch some of the talks, this is possible. Please ask them to set up a profile on the IOVC website and register for “Online viewing of conference talks” under Conference registration & abstract submission (this is a free registration). The link to the talks will then appear in Other Materials in My Congress Materials as described above.


Please note that the video will be available until 11 May for streaming only.

Photos from the conference are available on Flickr. More pictures will be added over the coming weeks as they are still coming in, so do check back!

Spring Symposium 2018 – Netherlands Society of Gene and Cell Therapy

The NVGCT Spring Symposium that was held on March 15 and 16, 2018 at conference center De Werelt in Lunteren was a great success. A total of 21 scientific presentations on gene and cell therapy related topics were given by young scientists and internationally renowned invited speakers, including Graziella Pellegrini, Marcel Tijsterman, Sander van Deventer and Ben Berkhout

Source: Spring Symposium 2018 – Netherlands Society of Gene and Cell Therapy