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Eli Lilly and Company has expanded a licencing and partnership agreement with ProQR Therapeutics to discover, develop and market new genetic medicines. The post Lilly and ProQR to expand genetic medicine development agreement appeared first on Pharmaceutical Technology.
CF is a progressive genetic disease caused by defective CFTR proteins, which are crucial for regulating salt and water movement in cells. These mutations can vary in severity and impact on CFTR function, from complete protein dysfunction to defects in protein folding, trafficking or regulation.
Collaborative research led by investigators at Dana-Farber/Boston Children's Cancer and Blood Disorders Center defines a novel approach to understanding how certain proteins called transcription factors determine which genetic programs will drive cell growth and maturation. The study is published in the journal Science.
Last week, Nature magazine published a paper detailing an in-situ study of Parkinson’s disease (PD), showing protein structural changes in cerebrospinal fluid (CSF) between healthy individuals and Parkinson’s patients. In PD clinical research, many biomarkers, such as neurofilament, can be measured within the CSF using a lumbar puncture.
Researchers from Children's Hospital of Philadelphia (CHOP) and Princeton University have discovered a novel genetic disorder associated with neurodevelopmental differences. The study "Abrogation of MAP4K4 protein function causes congenital anomalies in humans and zebrafish" is published in Science Advances today, April 26.
Team of Japanese and European scientists identify a novel genetic mitochondrial disorder by analyzing DNA samples from three distinct families Credit: Fujita Health University DNA ligase proteins, which facilitate the formation of bonds between separate strands of DNA, play critical roles in the replication and maintenance of DNA.
Credit: Masato Kanemaki Researchers can now more accurately and precisely target specific proteins in yeast, mammalian cells and mice to study how knocking down specific protein traits can influence physical manifestation in a cell or organism. “Conditional gene knockout and small interfering RNA […].
Credit: UPMC PITTSBURGH, May 7, 2021 – In a paper published today in Nature Communications, an international group of collaborators led by researchers at UPMC Children’s Hospital of Pittsburgh have identified a genetic cause of a rare neurological disorder marked by developmental delay and loss of coordination, or ataxia.
Researchers at the National Institutes of Health and their colleagues have found that a toxic protein made by the body called DUX4 may be the cause of two very different rare genetic disorders.
These vaccines were developed using different “platform technologies” that involve slotting genetic material from the virus into a tried and tested delivery package. While such platform technologies are a non-traditional approach, that does not mean they are untested. The power of social media also came into play.
While there is no cure to this neurodegenerative condition, academics and companies are pushing through with research that could help patients and their families. Other lines of research look at the genetic overlap between FTD and amyotrophic lateral sclerosis (ALS), which could be used in the development of treatments for both conditions.
Biomedical and genetic engineers at Duke University and the Albert Einstein College of Medicine have designed a small fluorescent protein that emits and absorbs light that penetrates deep into biological tissue.
Russian researchers and their international collaborators have developed a full-function bioelectronic photocell using one molecule of a fluorescent protein attached to a carbon nanotube.
Historically, these indications have challenged the one-size-fits-all treatment approach due to patient variability, such as genetic differences in drug metabolism and underlying health conditions. This resistance may occur due to genetic mutations, epigenetic changes, or a tumor’s microenvironment.
Credit: Dr Jie Zheng An innovative genetic study of blood protein levels, led by researchers in the MRC Integrative Epidemiology Unit (MRC-IEU) at the University of Bristol, has demonstrated how genetic data can be used to support drug target prioritisation by identifying the causal effects of proteins on diseases.
Despite their clinical heterogeneity, these diseases often share overlapping pathological mechanisms, including protein misfolding and aggregation, synaptic dysfunction, neuroinflammation, and axonal degeneration. Alzheimers Disease AD is a progressive neurodegenerative disorder characterized by cognitive decline and memory impairment.
Researchers at City of Hope in the US have developed a new potential therapy to treat glioblastoma multiforme (GBM), a kind of brain cancer. For this approach, the team leveraged an oncolytic herpes simplex virus for releasing chemokines, which are signalling proteins that aid in attracting immune cells to tumours.
Credit: Surajit Chatterjee To better understand how RNA in bacteria gives rise to protein–and along the way, target these processes in the design of new antibiotics–researchers are turning their attention to the unique way this process happens in bacteria.
But several researchers and experts have brought to light some food safety concerns of plant-based meat, and why it must be treated differently than that of animal protein. Because of their near-neutral pH and high protein and moisture content, plant-based meats are susceptible to microbial growth. Subject to Microbial Growth.
Medical charity Alzheimer’s Research UK has teamed up with artificial intelligence specialist Exscientia to find new drug treatments for the devastating neurodegenerative disease. . It reckons its use of AI and machine learning can trim years off the current 12- to 15-year cycle from early research to marketed product.
Discussions of cancer often stress the genetic mutations that drive disease by altering the normal function of cellular proteins. KRAS, for example, normally acts as an on/off switch for cellular proliferation, but mutations to the gene—common in lung cancer, colorectal cancer and pancreatic cancer—cause that switch to stay on.
Mutations can disrupt protein binding through a “burr effect” thus interfering with the regulation of cell growth Credit: Kümmel team/Oeckinghaus team Tuberous Sclerosis Complex (TSC) affects between one and two of every 10,000 new-born babies.
Researchers from Drexel University have uncovered a novel regulatory mechanism in the brain that is essential for making the right kinds of proteins that promote healthy brain function, and its malfunctioning may be an early contributor of the development of Alzheimer's disease.
Researchers from Cologne, Bochum, Padova and Angers have discovered a novel connection between mitochondrial function, protein quality control and cellular health, whose failure could be the leading cause of the currently incurable neurological disease Charcot-Marie-Tooth (CMT).
Alternative Splicing is an extraordinarily complex process that requires the coordinated action of multiple proteins, each specialised in very specific functions. These proteins are assembled and matured, forming a kind of consortium of proteins that perform these gene reading functions.
Studies using human cell lines and tumors grown in mice provide early evidence that inhibiting RNA-binding proteins, a previously overlooked family of molecules, might provide a new approach for treating some cancers Credit: UC San Diego Health Sciences In cancer research, it’s a common goal to find something about cancer cells — some sort (..)
Genetic mutations, both germline and acquired, are behind a large proportion of the most debilitating and sometimes life-threatening human diseases. A new frontier in cancer research. But scientists have struggled to find effective treatments for many of these diseases since the dawn of modern medicine.
Mark Hurst just wanted to kill some bugs. A newly credentialed bacteriologist working for the New Zealand government in the early 2000s, his job was to invent new and exciting weapons in the war against grass grubs, the squishy, ectoplasmic beetle larvae that had long bedeviled the islands’ farmers.
Duchenne Muscular Dystrophy (DMD) is a genetic disorder that primarily affects males, caused by a mutation in the gene that encodes for a protein called dystrophin. This protein is crucial for the normal functioning of muscles, and its absence leads to progressive muscle weakness and wasting.
– Researchers in the Oregon State University College of Science have taken a key step toward new drugs and vaccines for combating COVID-19 with a deep dive into one protein’s interactions with SARS-CoV-2 genetic material. Credit: OSU College of Science CORVALLIS, Ore.
Wednesday 19 May 2021 – New research published today sheds important light on how the production of a key protein in the brain is controlled, which could pave the way for new treatments for a wide range of neurological conditions.
The technology platforms include the Life Edit gene editing platform, an RNA, cell, protein, vector engineering and induced pluripotent stem cells (iPSCs) platform. ElevateBio intends to use the funds to advance its genetic medicine current good manufacturing practice (cGMP) and process development business, BaseCamp.
The modified contract is backed by previously allocated federal funding from Biomedical Advanced Research and Development Authority, HHS, and the Administration for Strategic Preparedness and Response. It was developed using the recombinant nanoparticle technology of Novavax.
Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Genetic study of proteins is a breakthrough in drug development for complex diseases.An
An international research team led by Dr. Ana Guadaño at the Alberto Sols Biomedical Research Institute (IIBM, a combined CSIC-UAM center) and involving the Complutense University of Madrid (UCM), used CRISPR gene editing techniques to incorporate into mice a mutation of the MCT8 protein responsible for transporting thyroid hormones to the interior (..)
A study by Indiana University School of Medicine researchers sheds new light on the development and treatment of a rare form of muscular dystrophy. The study's findings were recently published in Nature Communications.
Researchers in the US have developed an artificial intelligence-based tool that is able to predict COVID-19 symptoms and suggest which FDA-approved drugs might be used to treat patients. It could also be made more powerful by incorporating personal genetic information and gene expression profiles in tissues such as the lungs, they suggest.
MiNA Therapeutics has entered into a research collaboration and option licensing agreement with BioMarin Pharmaceutical to speed up the development of therapeutic ribonucleic acid activation (RNAa) candidates to treat rare genetic diseases. The new deal excludes oncology and other therapeutic areas outside the scope of genetic disease.
Researchers at the UNC School of Medicine have discovered that hepatitis A virus (HAV) replication needs particular interactions between the human protein ZCCHC14 and TENT4 poly(A) polymerases, a group of enzymes. In addition, the scientists later found that the HAV needs TENT4A/B for its replication.
Findings explain how plants use RNA to defend against fungal invaders Credit: Nicole Ward Gauthier/University of Kentucky New research reveals an essential step in scientists’ quest to create targeted, more eco-friendly fungicides that protect food crops.
ImmunoForge has expanded its licence agreement with Duke University in North Carolina, US, to jointly improve research and drug development opportunities. ImmunoForge chief technology officer Dr Jim Balance pioneered the clinical development of peptide drugs genetically fused to ELPs.
Studying mice, researchers at Washington University School of Medicine in St. Louis have found that proteins made by stem cells that regenerate the cornea may be new targets for treating and preventing such injuries.
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