Hereditary Diseases Articles
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Preclinical Animal Models as A Resource to Explore Rare Diseases
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 ...
By Protheragen
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Comprehensive Insights into Native Porcine Pancreatin: A Versatile Enzyme Preparation
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 ...
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The Sophisticated Mechanism of Epigenetic Induction Facilitating Cellular Proliferation Service
The intricate nature of cellular growth and proliferation has been a fascinating subject of study for cell biologists for many decades. Recently, with the advent of cutting-edge gene manipulation and control techniques, increasing emphasis has been laid on comprehending the role of epigenetic induction in facilitating cell growth. This article focuses on shedding light on the ingenious process of ...
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Gene Therapy for Cystic Fibrosis
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 ...
By Protheragen
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The Development Process of DUBTAC
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. ...
By BOC Sciences
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Clinical CRISPR Success Demonstrates Need for Quality Control
The Clinical CRISPR Successes Are Stacking Up. But Where’s the Emphasis on Quality Control? In mid-September, Intellia released some promising data on two of their CRISPR-based therapeutics. These results are a significant milestone for genome editing and validation of its effectiveness in the clinic. However, the safety of CRISPR-based therapeutics has been a major concern and part of a ...
By CRISPR QC
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Open-Source AI Platforms: Driving Success through Diversity
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 ...
By AiCure
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Mitigating Bias in AI: Holding Innovation to a Higher Standard
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 ...
By AiCure
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World Rare Disease Day
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 ...
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Benefits of point of care PFT in Rural Health
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 ...
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Recapitulating the Multifaceted Inflammatory Environment of the Intestine In Vitro
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 ...
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Enhancing Human Gut Tissue Models through Gene Editing
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 ...
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VIC Portfolio Companies and Pipeline Technologies with Pandemic Impact
Our current daily activities bear little resemblance to the routines as of as recently as a month ago. COVID-19 has had an obvious and significant impact on the American people. For those suffering from chronic conditions such as dementia, diabetes, heart and respiratory disease, the health impact during traumatic periods such as this is magnified. The COVID-19 pandemic has disrupted our ...
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