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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.
Discover the groundbreaking patent by Yield10 Bioscience Inc for a genetically engineered oilseed plant expressing a plant protein, revolutionizing biotechnology in agriculture.
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.
A team of Gladstone and UCSF scientists used a large-scale genetic approach to map the structure of protein complexes in live cells Credit: QBI, UCSF SAN FRANCISCO, CA–December 10, 2020–One of biologists’ most vexing tasks is figuring out how proteins, the molecules that carry the brunt of a cell’s work, do their job.
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.
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. The Japan-based team published their results on November 11th in Nature Communications.
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. Genetic biomarkers are being investigated to aid in the development of precision medicine for PD.
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.
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.
Antisense therapies are designed for destroying mRNA, reducing the amount of disease-causing protein. They will also have the potential to treat diseases that are caused by too little protein by increasing production, thus restoring the protein to normal levels.
Scientist are now combining recent advances in evolutionary analysis and deep learning to build three-dimensional models of how most proteins in eukaryotes interact.
A major challenge in human genetics is understanding which parts of the genome drive specific traits or contribute to disease risk. This challenge is even greater for genetic variants found in the 98% of the genome that does not encode proteins.
is revolutionizing adoptive cell transfer therapies with patented methods to reduce HLA-A protein expression in engineered human cells. Discover how Intellia Therapeutics Inc. Explore the potential for treating cancer, infectious diseases, and autoimmune disorders.
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.
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.
There’s a new kid on the block among companies using artificial intelligence in drug discovery, after 1910 Genetics launched today with $26 million in financing. 1910 Genetics is named after the year in which sickle cell disease (SCD) was first discovered in the US by James Herrick.
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.
Enhance protein expression and cellular targeting for effective treatment of genetic disorders. Discover Amicus Therapeutics' groundbreaking gene therapy vectors and methods of use. Explore the patent claim now!
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. This protein promotes lysosomal function. Proteins and biomarkers The pathologic buildup of proteins is also a potential drug target.
The protein-based vaccine is engineered from the genetic sequence of the SARS-CoV-2 virus’ initial strain. Developed utilising Novavax’s recombinant nanoparticle technology to generate antigen obtained from the coronavirus spike (S) protein, the vaccine is also formulated with the company’s saponin-based Matrix-M adjuvant.
A protein-based vaccine, NVX-CoV2373 is made from the genetic sequence of the SARS-CoV-2 virus’ first strain. Our protein-based vaccine was developed using an innovative approach to traditional technology and has demonstrated efficacy and safety in both adolescents and adults.”.
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. The […].
So we were excited when we were referred to a pediatric geneticist who offered Gabrielle whole exome sequencing , which sequences all of the protein-coding regions of the genome, to determine whether there was a genetic cause. Read the rest…
Alopecia is an autoimmune disorder that causes non-scarring hair loss on the scalp and body that is experienced by almost 2% of the global population at some point in their lifetime.
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.
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.
Despite their clinical heterogeneity, these diseases often share overlapping pathological mechanisms, including protein misfolding and aggregation, synaptic dysfunction, neuroinflammation, and axonal degeneration. Elevated NfL levels in CSF and serum correlate with disease activity, brain atrophy, and long-term disability.
Irregularities in the body's genetic coding to make proteins are linked to cancerous tumors. But most genetic material contains elements whose function isn't clear.
Administered as a single dose, the genetically engineered vaccine has the replication-defective adenovirus type 5 vector that expresses the spike S protein of the SARS-CoV-2 virus. In May, CanSinoBIO received an emergency use listing (EUL) from the World Health Organization (WHO) for its recombinant Covid-19 shot, Convidecia.
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.
Taldefgrobep is a complete human anti-myostatin recombinant protein that is developed for lowering free myostatin. According to Biohaven, SMA affects nearly one in 11,000 births in the US, and it is a genetic carrier in approximately one in every 50 Americans.
University of Utah Health scientists have corrected abnormal heart rhythms in mice by restoring healthy levels of a protein that heart cells need to establish connections with one another.
Eukaryotes (from the Greek meaning “true kernel”) have a cell nucleus that harbours most of the cell’s genetic information and includes organisms such as humans, plants and fungi. In prokaryotes, the cell’s contents, including its genetic material, are diffusely distributed. Eukaryotes typically have much larger […].
The genome also has the code for making a capsid, the protein shell of a virus that encapsulates the genome and protects it like a nanocontainer. . — Each simple RNA virus has a genome, its “native RNA.” This genome dictates how the virus replicates in cells to eventually cause disease. Credit: Zandi lab, […].
A protein-based vaccine, NVX-CoV2373 is created from the genetic sequence of the SARS-CoV-2 virus’ initial strain. It is formulated with the company’s saponin-based Matrix-M and also comprises a purified protein antigen.
An international study, coordinated by experts from the University of Nottingham, has revealed that the genetic risk of pre-eclampsia – a potentially dangerous condition in pregnancy – is related to blood pressure and body mass index. It […].
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).
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 (..)
A University of Massachusetts Amherst scientist has discovered that a mutation in the misfolding protein that causes Parkinson's disease offers protection against another fatal neurodegenerative disorder—multiple system atrophy (MSA), which is caused by the same misfolding protein.
Eloxx is conducting a Phase II trial (NCT05448755) investigating ELX-02 in the treatment of Alport syndrome – a genetic disorder characterised by kidney disease. ELX-02 is a small molecule drug designed to increase the probability full-length proteins are produced by promoting ribosomal function.
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