OnTarget: in silico design of MiniPromoters for targeted delivery of expression

Abstract MiniPromoters, or compact promoters, are short DNA sequences that can drive expression in specific cells and tissues. While broadly useful, they are of high relevance to gene therapy due to their role in enabling precise control of where a therapeutic gene will be expressed. Here, we present OnTarget (http://ontarget.cmmt.ubc.ca), a webserver that streamlines the […]

2023 Gairdner High School Symposium

Student Registration All Grade 10-12 students are eligible to participate in the Gairdner High School event. Registration is required. Before registering you will need to: Method of Participation In-person Monday, October 23 | 8:45 am – 12:15 pm Arrange to miss class and plan your own transportation to BC Children’s Hospital. You have the option […]

CMMT Newsletter July 2022

CMMT Newsletter July 2022 The Centre for Molecular Medicine and Therapeutics would like to welcome four new faculty members.   CMMT Newsletter July 2022 Welcome New Members! We have EXCITING news that we would like to share with you! The Centre for Molecular Medicine and Therapeutics (CMMT) would like to welcome four new faculty members. […]

2022 Vancouver Gairdner National Symposium

The Vancouver Gairdner High School & National Symposium is held each fall to give students, teachers, and the academic community the opportunity to hear from two Gairdner award-winning scientists and learn about the BC Children’s Hospital’s research facilities. Click here to watch a recording of the Gairdner High School Symposium Watch the 2022 Vancouver Gairdner […]

2021 Vancouver Gairdner National Symposium Recordings/VOD

These are the recordings of the 2021 Vancouver Gairdner Symposium, held on Oct 25, 2021. High School Symposium – Morning Session UBC Gairdner Symposium – Afternoon Session

Simpson Lab paper in Nature’s Gene Therapy

Read the new paper from the Simpson lab in Nature’s Gene Therapy: Human MiniPromoters for ocular-rAAV expression in ON bipolar, cone, corneal, endothelial, Müller glial, and PAX6 cells, here: https://rdcu.be/ceDDL

Gairdner Highschool Symposium: Trainee Event

The Gairdner High School Symposium is a half-day event held each fall that gives secondary students and teachers the opportunity to hear from Gairdner award-winning scientists and attend a behind-the-scenes tour of BC Children’s Hospital’s research facilities. This event is hosted in conjunction with the Center for Molecular Medicine and Therapeutics (CMMT) and the Canada Gairdner Foundation, recognizing the world’s most creative and […]

2020 Gairdner Highschool Symposium: Morgan Towriss – Mental Health

This is the recording of the Morgan Towriss, Honours Thesis Student – Jan Lab. Please enter any questions you may have for Morgan below and we will get back to you as soon as we can.

2020 Gairdner Highschool Symposium: Vaishnavi Sridhar – Cells

This is the recording of the Vaishnavi Sridhar, PhD Student – Conibear Lab. Please enter any questions you may have for Vaishnavi below and we will get back to you as soon as we can.

Dr. Wyeth Wasserman among four UBC faculty named AAAS Fellows in 2020

This year, four UBC faculty members have been elected as Fellows of the American Association for the Advancement of Science (AAAS), the world’s largest multidisciplinary scientific society and publisher of the Science family of journals. Jesse Brewer (Physics): For his pioneering work developing muSR, in which muons are used to probe quantum materials, with critical […]

Re-writing the code of life: GenomeBC podcast

Two scientists recently shared the Nobel Prize for Chemistry for transforming an obscure bacterial immune mechanism, called CRISPR, into “genetic scissors” – an editing tool for re-writing the code of life. The two female Nobel Prize winners accomplished this through collaboration – which is exactly how two Canadian scientists – Dr. Elizabeth Simpson (CMMT) and […]

CMMT Newsletter September 2020

Changing the World: It Pays to Dream In 1983, Dr. Michael Hayden arrived at the University of British Columbia (UBC) with a big idea, some might even say a dream. A physician-scientist with a focus on genetic diseases, particularly Huntington’s Disease and lipoprotein disorders, Dr. Hayden sought to establish a research centre that embraced an […]

New antivirus options from UBC IT

As of 2020 July, UBC IT has mandated a changeover in cyber security/anti malware software. Here is what you need to know: Your on-site desktop computers that log in with CMMT network credentials will be handled by CMMT IT Your UBC-owned laptops will need you to do the following – Uninstall SOPHOS (see section below) […]

Dr. Elizabeth Simpson Awarded Fighting Blindness Research Grant

Using gene therapy to treat congenital blindness Aniridia is an eye disorder where the iris, the coloured part of the eye, is partially or completely absent. Individuals with aniridia usually have low vision from birth and develop glaucoma and cataracts, which can lead to blindness. Current treatments may slow the progression of aniridia, but there […]

Leadership Update: Bruce Verchere appointed Director, Centre for Molecular Medicine and Therapeutics

        Dear Colleagues, It is with great pleasure that we announce the appointment of Dr. Bruce Verchere as the Director of the Centre for Molecular Medicine and Therapeutics (CMMT).                 Dr. Verchere is a highly accomplished researcher and Professor in the Departments of Pathology and […]

2019 International Gairdner Symposium

The Gairdner International Symposium is held each fall to give students, teachers, and researchers the opportunity to hear from Gairdner award-winning scientists and attend a luncheon with the award winners. Watch the video of the 2019 Gairdner High School Symposium: HERE Watch the video of the 2019 Gairdner National Symposium: HERE Time: 3:30- 6:00 pm, Monday, October 21, 2019 […]

CMMT researchers mining 25 years of data uncovers a new predictor of age of onset for Huntington disease

May 16, 2019 Study lead author Galen Wright Investigators at the University of British Columbia (UBC) Centre for Molecular Medicine & Therapeutics (CMMT) and BC Children’s Hospital have examined more than 25 years of data to reveal new insights into predicting the age of onset for Huntington disease. “This discovery may enable us to provide […]

Huntington drug successfully lowers levels of disease-causing protein

An international clinical trial has found that a new drug for Huntington disease is safe, and that treatment with the drug successfully lowers levels of the abnormal protein that causes the debilitating disease in patients. In the study published today in the New England Journal of Medicine, researchers from UBC and their colleagues have demonstrated […]

Huntingtin Lowering Strategies for Disease Modification in Huntington’s Disease

Huntington’s disease is caused by an abnormally expanded CAG repeat expansion in the HTT gene, which confers a predominant toxic gain of function in the mutant huntingtin (mHTT) protein. There are currently no disease-modifying therapies available, but approaches that target proximally in disease pathogenesis hold great promise. These include DNA-targeting techniques such as zinc-finger proteins, transcription activator-like effector nucleases, […]

Trainee Spotlight: Jeffy Rajan

Kudos to Jeffy Rajan, recipient of a 2019 Brain, Behaviour and Development (BB&D) Trainee Boost Award.  Jeffy hangs out in the Goldowitz Lab and her project is “Impact of motor learning performance in Developmental Coordination Disorder (DCD)”. “I am very grateful to be the recipient of the BB&D Trainee Boost Award in recognition of a […]

Lowering levels of mutant protein that causes Huntington disease can restore cognitive function in mice

Lowering levels of mutant protein that causes Huntington disease can restore cognitive function in mice

2018 International Gairdner Symposium

The Gairdner High School Symposium is a half-day event held each fall that gives secondary students and teachers the opportunity to hear from Gairdner award-winning scientists and attend a behind-the-scenes tour of BC Children’s Hospital’s research facilities. Watch the video of the 2018 Gairdner High School Symposium: HERE Time: 3:30- 6:00 pm, Monday, October 22, 2018 Venue: LSC2, Life […]

Strict eating schedule can lower huntington disease protein in mice

Strict eating schedule can lower Huntington disease protein in mice

BC and UK partnership to tackle rare diseases through genomics

Vancouver, BC — A new partnership pilot project between Genome British Columbia (Genome BC) and Genomics England will focus on improving the diagnosis of rare diseases in children while furthering the discovery of new, novel diseases in BC and around the world. More than 80 per cent of the 7000 known rare diseases are genetic […]

‘Phenomenal’ trial results may lead to a treatment for Huntington’s disease, experts say

via the Washington Post: The discovery of a drug that may treat the fatal disease known as Huntington’s is being hailed as “historic” by Louise Vetter, president and CEO of the Huntington’s Disease Society of America, and “phenomenal” and “fantastically promising” by Huntington’s researchers, including the woman who discovered the genetic mutation that causes the disease. “I’m […]

Ionis Antisense Oligonuleotide Drug IONIS-HTTRX is Safe and Decreases Levels of Toxic Huntington’s Disease Protein in Cerebrospinal Fluid.

In a statement released today, Ionis Pharmaceuticals announced study results demonstrating for the first time that their drug (IONIS-HTTRX) lowered levels of the abnormal protein causing Huntington’s disease in cerebrospinal fluid, and that this approach was safe and well tolerated in humans. Link: http://ir.ionispharma.com/news-releases/news-release-details/ionis-pharmaceuticals-licenses-ionis-htt-rx-partner-following   The IONIS-HTTRx Study: The IONIS-HTTRX trial enrolled 46 patients with […]

Potential Breakthrough for Huntington’s Disease

Lancet, Neurology paper released “Neurofilament light protein in blood as a potential biomarker of neurodegeneration in Huntington’s disease: a retrospective cohort analysis”. Authors: Lauren M Byrne, MRes, Filipe B Rodrigues, MD, Prof Kaj Blennow, PhD,  Prof Alexandra Durr, PhD, Prof Blair R Leavitt, PhD, Prof Raymund A C Roos, PhD, Rachael I Scahill, PhD, Prof […]

Pope embraces Huntington’s afflicted in bid to end stigma

http://www.ctvnews.ca/health/pope-embraces-huntington-s-afflicted-in-bid-to-end-stigma-1.3418951 VATICAN CITY — Pope Francis embraced weeping mothers, fathers and children with Huntington’s Disease on Thursday as he sought to remove the stigma of an incurable genetic disorder that causes such devastating physical and psychiatric effects that its sufferers are often shunned and abandoned. One by one, Francis blessed and greeted each of the […]

Dr. Michael R. Hayden Inducted into the Canadian Medical Hall of Fame

On Thursday, May 4th, six renowned medical pioneers will be recognized as the 2017 Canadian Medical Hall of Fame Inductees at a special ceremony in Québec City, hosted by Université Laval and its Faculty of Medicine and sponsored by the Canadian Medical Association and MD Financial Management. “The Canadian Medical Hall of Fame is proud […]

BC Children’s Hospital researchers & UBC students create their own e-textbook and new software platform

Undergraduate students in Dr. Wyeth Wasserman’s cancer genetics class had a unique assignment: to write their own e-textbook for their course. “Textbooks cost too much and students should have high quality, free materials,” says Dr. Wasserman, who led the project. He is executive director of BC Children’s Hospital Research Institute, professor in the Department of […]

Dr. Michael S. Kobor appointed as the Sunny Hill BC Leadership Chair in Child Development

Congratulations to Dr. Michael S. Kobor Dr. Michael S. Kobor  has been appointed as the Sunny Hill BC Leadership Chair in Child Development. The Chair has a $6-million endowment that will support research on understanding how early life experiences affect gene expression to influence children’s health and development. Dr. Kobor seeks to understand why some […]

October 6, 2016 – Dr. Michael Hayden inducted into the Canadian Medical Hall of Fame

October 7th, 2016 Vancouver, B.C. [October 6, 2016] Dr. Michael Hayden, Killam Professor of Medical Genetics at the University of British Columbia and Canada Research Chair in Human Genetics and Molecular Medicine, will be inducted into the Canadian Medical Hall of Fame. He joins five other outstanding doctors whose outstanding contributions to health have made […]

June 22, 2016 – Is Huntington’s Disease More Common than We Thought?

June 22, 2016 – More people may have the potential to develop Huntington’s disease than previously thought, according to a study published in the June 22, 2016, online issue of Neurology®, the medical journal of the American Academy of Neurology. But the increase comes in the percentage of people who have a lower risk of […]

A handful of British Columbians are helping test a new “gene silencing” drug designed to halt production of a toxic protein that causes brain wasting in Huntington Disease.

October 21st, 2015 A handful of British Columbians are helping test a new “gene silencing” drug designed to halt production of a toxic protein that causes brain wasting in Huntington Disease. A clinical trial now underway to prove the safety of the experimental drug ISIS-HTT will include 36 patients with very early symptoms of the […]