Cystic Fibrosis Related Diabetes Published In The Journal Of Cystic Fibrosis Articles & Analysis
15 articles found
ELANE, or neutrophil elastase (NE), is a serine protease stored in the azurophilic granules of neutrophils and is encoded by the ELANE gene on chromosome 19. It plays a pivotal role in the host defense system by degrading bacterial proteins, remodeling extracellular matrices, and shaping inflammatory responses. However, the same proteolytic activity that protects against pathogens can, when ...
Gene delivery is a therapeutic strategy that’s revolutionizing biotechnology and pharmaceuticals. It involves the delivery of new genetic molecules into target cells to treat diseases. This technology has the implicit to revise the treatment of inherited genetic disorders, cancer and other intractable driseases. Scientists are using a variety of gene delivery strategies to insert new ...
Elastase is a powerful enzyme that plays a critical role in various biological processes, both in physiological and pathological contexts. It belongs to the family of serine proteases, which are enzymes characterized by a unique serine residue in their active site. This enzyme is known for its ability to degrade elastin, a key protein that provides elasticity and resilience to tissues such as ...
What is biofilm? Biofilms are defined as communities of micro-organisms attached to a surface, or one another, and encased within a matrix of extracellular polymeric substance (EPS). The EPS makes up the largest component of the biofilm, and in the biological environment is generally composed of polysaccharides, proteins, glycolipids, blood products, cellular debris, extracellular enzymes, metal ...
Rare diseases, also known as orphan diseases, are a diverse group of disorders that affect a small percentage of the population. Due to their low prevalence and limited understanding, treating and finding cures for these conditions present significant challenges. However, preclinical animal models have emerged as invaluable resources for advancing research and unraveling the mysteries surrounding ...
Native Porcine Pancreatin, an enzymatic mixture derived from the porcine pancreas, has emerged as a fundamental component in various industries due to its unique enzymatic composition. Comprising amylase, lipase, and protease enzymes, this preparation is celebrated for its multifaceted applications and plays an indispensable role in the pharmaceutical, food, and biotechnology sectors. In this ...
What is cystic fibrosis? Cystic fibrosis (CF) is a rare genetic disorder that affects the lungs, digestive system, and other organs. It is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which encodes a protein that regulates chloride ions transport across cell membranes. These mutations cause the CFTR protein to be either absent or not function ...
The use of Proteolysis Targeting Chimeras (PROTACs) to degrade dysfunctional proteins has shown great potential to target proteins that were previously considered “undruggable”, and several projects have entered clinical evaluation worldwide. Aberrant protein degradation, in fact, is also a pathogenic mechanism in some diseases, such as cystic fibrosis (CF) and some forms of cancer. ...
Many cell and gene therapy solutions have shown considerable promise as a source of possible treatments for a wide range of chronic disorders. However, these sophisticated therapies introduce additional barriers such as the requirements for ultra-low and cryogenic storage, which affect numerous steps of the biomedical solutions’ life cycles, from their development to their administration. ...
Digital biomarkers can open a world of possibilities in understanding the nuances of a patient’s behavior and response to treatment. Rather than relying on subjective perceptions of how a patient is responding, digital biomarkers treat patient observation more like an engineering problem. Such precise, sensitive measurements can ultimately influence the future of a patient’s care plan ...
ByAiCure
When it comes to building equitable, quality AI, prioritizing diverse data sets needs to be embedded in a developer’s DNA – rather than a “nice to have,” it should be a deliberate framework in which AI is built. Before deploying an algorithm to patients, it should be rigorously evaluated under both common and rare scenarios to ensure it performs as intended and ...
ByAiCure
On February 29 (or the last day of February) we celebrate World Rare Disease Day to raise awareness about these diseases, aiming to improve access to diagnosis, treatment and medical care. What are these diseases? Rare diseases are those that occur infrequently in the general population. In Argentina, “rare diseases” are those whose prevalence in the general population is equal to ...
There’s an island in northern Lake Michigan called Beaver Island. It sits roughly 30 miles offshore of Michigan’s Lower Peninsula, part of an archipelago of other whimsically named islands (including Whiskey, Hog, Garden, and Hat). Roughly 500 hardy souls call Beaver Island home year-round, and the two townships that occupy the land support a small but stable economy that embodies the ...
Intestinal inflammatory diseases are highly complex, which has thus far prevented development of an effective in vitro disease model. With access to a donor-derived cell biobank and the ability to recapitulate region-specific environments of the intestine, Altis Biosystems is working toward the development of an in vitro RepliGut® model to accurately recapitulate the complex intestinal ...
The variety of normal and diseased human tissues needed to perform comprehensive screening of drug candidates is a key resource for next-generation preclinical models, but obtaining these tissues is challenging. Gene editing enables Altis Biosystems to offer a diverse array of RepliGut® human gut models tailored for specific research programs. Many Challenges Around Accessing Tissues ...