Larry Lands, a senior scientist at The Institute, will lead a new project focusing on rare genetic mutations
SOURCE: Montreal Children’s Hospital
March 17, 2025
A new research project focusing on rare genetic mutations linked to cystic fibrosis just received $600,000 in funding over three years from Génome Québec and Cystic Fibrosis Canada. Led by Larry Lands, MD, PhD, Senior Scientist in the Translational Research in Respiratory Diseases (RESP) Program at the Research Institute of the McGill University Health Centre (The Institute), and Jacques-P. Tremblay, Professor at Laval University, the study targets rare orphan mutations in the CFTR protein for which there is no treatment option.
While there are therapies that improve the function of the defective CFTR protein that causes cystic fibrosis, these therapies do not work in people who have rarer genetic mutations that fail to produce CFTR proteins, leaving them without the life-changing treatment options that others with more common mutations can access. Genetic profiling of cystic fibrosis patients reveals that several of these rare mutations are especially prevalent in Quebec. Cystic fibrosis is the most common, fatal genetic disease affecting children and young adults in Canada.
The project “Leave No One Behind: development of a genetic therapy for people living with cystic fibrosis who do not respond to available modulators” proposes using a gene modification technique called prime editing to permanently correct mutations in the CFTR protein. The research team will explore ways to deliver prime editing by injection or inhalation of lipid nanoparticles to correct CFTR gene mutations in the airway cells of the lung.
In the lab, a cell model will be created using a common Canadian CFTR gene mutation unresponsive to available modulators. This model will serve as the first step in testing the effectiveness of the prime editing therapy. The team will also assess the therapy using a variety of methods, including cells from people with cystic fibrosis, to identify optimal ways of delivering it.
“This work could pave the way for clinical trials, offering hope to those people with cystic fibrosis whose mutations currently lack effective treatments,” says Dr. Lands, also Director of the Respiratory Medicine / Pulmonology Division at the Montreal Children’s Hospital.
This project brings together a unique multidisciplinary team with diverse backgrounds and experiences, including Christine Bear (Senior Scientist, SickKids), Danuta Radzioch, PhD, (senior scientist, Infectious Diseases and Immunity in Global Health (IDIGH) Program, The Institute), Darcy Wagner, PhD, (senior scientist, RESP Program, The Institute), Jun Ding, PhD, (junior scientist, RESP Program, The Institute), Christine DeWolf (Chemistry and Biochemistry, Concordia University), Nathan Luedtke (Chemistry, McGill University) and Ajitha Thanabalasuriar (Pharmacology and Therapeutics, McGill University).
One honor awaits another
Dr. Lands was recently awarded the King Charles III Coronation Medal, nominated by the Canadian Lung Association for his work on modulation of inflammation and exercise limitation in cystic fibrosis.
Congratulations Dr. Lands!