In a new study entitled “Natural Variation in Gene Expression Modulates the Severity of Mutant Phenotypes,” researchers show that disease severity in genetic disorders such as Cystic Fibrosis depends on the genetic background of individuals and not solely on the genetic defect of a particular gene. These…
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Children’s Healthcare of Atlanta, Emory University, and Georgia Tech were recently awarded a grant by The Cystic Fibrosis Foundation to expand the Atlanta CF Research and Development Program (CF@LANTA RDP Program). The four-year grant worth $1.8 million will enable the collaborating institutions to expand their current cystic fibrosis research projects and translate…
A new study recently published in the journal Nature Biotechnology reported the creation of miniature bile ducts in the lab and their use in the discovery of a new promising therapeutic drug for cystic fibrosis (CF). The study is entitled “Cholangiocytes derived from human induced pluripotent…
The Cystic Fibrosis Foundation, the world’s most prominent organization dedicated to the search for a cure for cystic fibrosis, has just announced its President and Chief Executive Officer, Robert J. Beall, Ph.D., will be stepping down from his position of 21 years. The current Executive Vice President for medical affairs, Dr. Preston…
Thiocyanate is a molecule found throughout the human body. Recent studies in animals have shown that thiocynate could be used as a treatment for lung inflammation, although more research is needed. These new insights are particularly important for the CF community, as thiocynate could potentially be useful as a treatment for…
Last week, the Her Royal Highness the Countess of Wessex, Sophie Rhys-Jones, officially inaugurated the Wolfson Cystic Fibrosis Center at Nottingham City Hospital. During the opening day, HRH made time to meet with CF patients, as well as the center’s staff and key donors. The new 250-bed center is estimated to be…
Gene therapy in the context of treating cystic fibrosis made a significant step forward with the announcement of positive data from a Phase IIb clinical trial investigating the VGXI-manufactured aerosolized DNA plasmid expressing Cystic Fibrosis Transmembrane Conductance Receptor (CFTR) — an experimental CF therapy that improved a key lung function…
Spyryx Biosciences, Inc., a private bio-pharmaceutical firm advancing new therapeutics to address obstructive lung diseases, recently announced it has received an award from Cystic Fibrosis Foundation Therapeutics that will provide support to the development of Spyryx’s therapeutic peptides to address cystic fibrosis. “We appreciate the generous support of the Cystic Fibrosis…
A study recently published in the journal The Lancet Respiratory Medicine revealed encouraging results of a phase 2b clinical trial assessing the safety and efficacy of a non-viral gene therapy based on a gene defective in cystic fibrosis (CF) patients. The study is entitled “Repeated…
A new study revealed that microRNA16 — a small non-coding RNA genes that regulates gene expression — can restore the F508del-CFTR protein function in airway cell lines and primary cultures of differentiated human bronchial epithelia from F508del homozygotes that express mutant CFTR protein endogenously. The finding is significant, as…
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