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How Organ Transplant Works

The misconception that all patients in need of organ donation are responsible for their ill health should not suffice. Of course, there are instances that people smoke or take alcohol to the extremes, leading to the failure of these vital organs. But this is not usually the scenario for all organ damage cases. Why? Sometimes, genetics are to blame. A good example is in the case of cystic fibrosis. Good news however is, donor transplant can alter the sad situation. Donation may come from a living person, or from a patient declared medically brain-dead. For a living donor, donation is only possible if survival is not compromised by the absence of one of the organs, like part of the liver or on

Common virus could speed up cystic fibrosis

A new study has found that cytomegalovirus, which is normally dormant in people who have it, can become active again in people with cystic fibrosis who develop lung infections. Cystic fibrosis is an inherited condition. It causes damage to the lungs and digestive system, and it is life-threatening. Common symptoms of cystic fibrosis include persistent coughing, inflamed nasal passages, wheezing, breathlessness, severe constipation, and lung infections. Cystic fibrosis occurs due to a gene mutation that affects the protein that regulates the movement of salt in and out of cells. The mucus, which is normally thin and slippery, becomes sticky and thick and clogs the tubes that carry air in and

Cytomegalovirus – an unrecognised potential contributor to cystic fibrosis disease progression?

Given increasing body of work supporting an association of cytomegalovirus (CMV) seropositivity with several chronic medical conditions, including heart disease and dementia, possibly associated with the impacts of chronic inflammation, researchers performed a single-center retrospective study to investigate whether the outcomes of cystic fibrosis (CF, a chronic inflammatory disease) patients could likewise be related to CMV-status. They analyzed 71 people with CF referred for lung transplantation and evaluated how CMV serostatus was related to patient pre-transplant outcomes. Findings revealed an association of CMV IgG positivity with disproportionate progression to end-stage lung disease,

Large Sugar Molecule Disrupts B. cepacia Bacteria from CF Patients, Study Shows

An experimental large sugar molecule called poly (acetyl, arginyl) glucosamine, or PAAG, was able to disrupt treatment-resistant biofilms formed by Burkholderia cepacia complex bacteria extracted from patients with cystic fibrosis (CF), according to a study. The study, “In Vitro Activity of a Novel Glycopolymer against Biofilms of Burkholderia cepacia Complex Cystic Fibrosis Clinical Isolates,” was published in the journal Antimicrobial Agents and Chemotherapy. Burkholderia cepacia complex (Bcc) is a strain of opportunistic bacteria that can cause lower respiratory infections in patients with CF. They often appear late in CF progression and form biofilms (layers of bacteria sticking to surfa

The Future of Cell and Gene Therapy

Each passing year, the success of cell and gene therapy (CGT) becomes clearer, more widely covered in the media, and is increasingly the focus of a rapidly growing society of researchers. Making sense of this extensive ecosystem is no small feat, but by using a data-driven approach, we can get closer to determining what the future holds for CGT. CGT has emerged as one of the most exciting areas of biotechnology. Its buzz can be attributed to the hope these therapies hold for patients with rare and often deadly inherited diseases; the scientific promise and intrigue of gene editing; and the business opportunity that these revolutionary therapies with lucrative price tags hold. But what do we

Abeona Therapeutics Details Pathway for Advancing Lead Clinical Programs and Unveils New Cystic Fibr

EB-101 pivotal trial for Recessive Dystrophic Epidermolysis Bullosa planned for mid-2019 enrollment Expanding Phase I/II study of ABO-102 for Sanfilippo syndrome type A (MPS IIIA) Novel AIMTM AAV vector with CFTR minigene addresses all mutations of Cystic Fibrosis NEW YORK and CLEVELAND, Dec. 06, 2018 (GLOBE NEWSWIRE) — Abeona Therapeutics Inc. (Nasdaq: ABEO), a leading clinical-stage biopharmaceutical company focused on developing novel cell and gene therapies for life-threatening rare genetic diseases, today announced key pipeline updates during the Company’s 2018 R&D Day. “The important clinical and preclinical updates we shared today further establish Abeona’s pathway to bring long-term

Verona Pharma Granted Key EU Patent Related to Late-stage COPD Clinical Candidate Ensifentrine

LONDON, April 09, 2019 (GLOBE NEWSWIRE) -- Verona Pharma plc (AIM:VRP) (Nasdaq:VRNA) ("Verona Pharma"), a clinical stage biopharmaceutical company focused on respiratory diseases, is pleased to announce that the European Patent Office has recently granted an additional key patent relating to its lead development candidate, ensifentrine. The patent provides intellectual property protection throughout Europe out to 2035 for a suspension formulation of ensifentrine (RPL554) suitable for nebulized administration. A corresponding patent has already issued in the US. Ensifentrine is in Phase 2b clinical trials for chronic obstructive pulmonary disease (COPD). This first-in-class inhaled therapy ha

Choline Supplementation Improves Lung Function, Liver Health in CF Patients, Study Shows

Choline supplementation improves lung function and liver health in people with cystic fibrosis (CF), and should be considered as a complementary treatment to current standard care therapies, a study suggests. The findings of the study, “Choline Supplementation in Cystic Fibrosis — The Metabolic and Clinical Impact,” were published in the journal Nutrients. CF is a genetic disorder caused by mutations in the CFTR gene, which provides instructions to make the cystic fibrosis transmembrane conductance regulator (CFTR) protein. This protein works as a channel that transports in and out of cells electrically charged molecules, such as chloride, which will have a direct impact on the regulation of

Mucus and Inflammation, Rather Than Infections, are Early Drivers of Lung Disease in CF, Study Says

Early accumulation of abnormally thick mucus and inflammation in the lungs, rather than bacterial infections, may be the initial trigger of lung disease during cystic fibrosis (CF), a new study suggests. Bacterial infections have been thought of as an early driver of CF disease, but mucus build-up and inflammation of the airways is already evident early in children with CF, despite little or no signs of infection. The results suggest that timely treatment of CF children with mucolytics — agents that help dissolve the mucus and make it more fluid — and anti-inflammatory therapies may delay the onset of lung disease and worse CF symptoms, potentially improving their life expectancy. The study

Positive Expiratory Pressure (PEP)

Cystic fibrosis (CF) is a genetic condition caused by abnormalities in the function of the cystic fibrosis transmembrane regulator (CFTR) protein within the mucus-producing cells of the body. This dysfunction compromises the transport of water and charged ions across the cell membrane and results in the production of thick and sticky mucus. The presence of sticky mucus in the airways can prevent the clearance of pathogens from the lungs and result in chronic respiratory obstruction. Removing this mucus is important to prevent infections in the airways, making it easier for CF patients to breathe. Common airway clearance techniques Chest physiotherapy is instrumental to clear mucus from clogg

Virus that Infects Bacteria Turns Immune Response Away from P. aeruginosa, Study Shows

Pseudomonas aeruginosa, the most common bacteria found in cystic fibrosis (CF) patients, uses a virus to go unnoticed by the host immune system, a new study shows. Treatment with a vaccine designed to help the immune system detect the virus was found to significantly reduce P. aeruginosa’s ability to infect mice, suggesting it might represent a new way to treat CF patients. The study, “Bacteriophage trigger antiviral immunity and prevent clearance of bacterial infection,” was published in the journal Science. P. aeruginosa, dangerous for CF patients, is also common in diabetic ulcers, bedsores, and burn wounds. At the moment there is no approved vaccine against these bacteria, and they are i

Developing a first-in-class regenerative gene therapy for cystic fibrosis

OmniSpirant Limited is an Irish company developing a regenerative gene therapy with the potential to transform respiratory medicine. The innovative OmniSpirant platform is based on the use of engineered stem cell exosomes as regenerative carriers for inhaled gene therapy. Exosomes are nanosized vesicles that are naturally produced by virtually all cells and are involved in cell-to-cell communication. Large volumes of recent research highlight the vast potential of stem cell exosomes as transformative regenerative gene therapy and medicines. The engineered exosome platform will initially be used to develop a first-in-class regenerative gene therapy for cystic fibrosis. OmniSpirant’s patent-pe

Symdeko Approved in Australia for CF Patients Age 12 or Older With Certain Gene Mutations

Cystic fibrosis (CF) patients in Australia age 12 and older, who have two copies of the F508del mutation in the CFTR gene (a copy inherited from each parent), now can be treated with Vertex Pharmaceuticals’ Symdeko (tezacaftor/ivacaftor and ivacaftor). The treatment also was approved for any mutation in the CFTR gene that responds to Symdeko based on lab tests and/or clinical evidence. Some of the treatment-responsive mutations are: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G, 3849+10kbC→T, E56K, R74W, D110E, D110H, E193K, E831X, F1052V, K1060T, A1067T, F1074L, and D1270N. CF patients who don’t know their gene mutation may want to co

Amphotericin B improves cystic fibrosis symptoms in cultured cells and pigs Antifungal drug forms io

An antifungal small molecule drug could restore some of the function lost in cystic fibrosis. Cystic fibrosis is a disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) protein, an ion channel that transports chloride, bicarbonate, and other anions across the membranes of epithelial cells lining lung airways. When the CFTR ion channel doesn’t work, it adversely affects the pH and viscosity of the liquid lining the airway surface and reduces an individual’s defenses against microorganisms. Martin D. Burke of the University of Illinois at Urbana–Champaign and coworkers added the drug, amphotericin B, to cultured epithelial cells from people with cystic f

Lung Enzyme Cathepsin S is New Therapeutic Target for CF and COPD

Blocking the enzyme cathepsin S can alleviate symptoms and reduce lung damage in cystic fibrosis (CF) and chronic obstructive pulmonary disease(COPD), according to multinational teams led by researchers at Queen’s University Belfast, U.K. The findings on CF were reported in the study “Targeting of Cathepsin S Reduces Cystic Fibrosis-like Lung Disease,” published in the European Respiratory Journal; the data on COPD was published in the American Journal of Respiratory and Critical Care Medicine, in an article titled “Protein Phosphatase 2A Reduces Cigarette Smoke-Induced Cathepsin S and Loss of Lung Function.” High levels of cathepsin S have been reported previously in the lungs of both CF an

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