Pharmaceutical Technology

JUNE 14, 2023

However, more immeasurable characteristics such as personality, behaviour, and even intelligence are all influenced by genetics to varying degrees. Despite its historic nature, the Human Genome Project had many setbacks, the greatest among them being the lack of genetic diversity. Each human cell has 1.8

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Genome Project Medicine Genomics Genome 130

Scienmag

APRIL 15, 2021

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.

Genetics 105

Genetics DNA Genomics Genome 105

Worldwide Clinical Trials

OCTOBER 14, 2024

Scientists now use cutting-edge techniques, including biological therapies that target specific immune system components and precision medicine approaches that personalize treatment plans based on a patient’s genetic profile.

Research 147

Research Clinical Trials Clinical Trials Immune Response 147

pharmaphorum

MARCH 26, 2021

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. Dr Jen Nwankwo.

Genetics 109

Genetics Drugs Protein Scientist 109

Worldwide Clinical Trials

OCTOBER 14, 2024

Scientists now use cutting-edge techniques, including biological therapies that target specific immune system components and precision medicine approaches that personalize treatment plans based on a patient’s genetic profile.

Research 130

Research Clinical Trials Clinical Trials Immune Response 130

STAT News

FEBRUARY 21, 2023

BEIJING — Hong Kong on Tuesday revoked a visa it granted to a Chinese scientist who set off an ethical debate five years ago with claims that he made the world’s first genetically edited babies, pulling it hours after he announced his research plans in the financial hub. Read the rest…

Scientist 98

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Scienmag

DECEMBER 23, 2020

Forward genetics pinpoints gene linked to ASD involving severe speech impairment and opens door to search for more mutations, future treatments Credit: Photo taken by Brian Coats for UT Southwestern Medical Center DALLAS – Dec.

Scientist 97

Scientist Gene Genetics Research 97

pharmaphorum

JANUARY 24, 2023

Ben Hargreaves finds that the vast amount of genetic data that exists today could help provide a faster, more targeted way of developing new drug candidates. The logical extension to this kind of approach is treating individual patients, with their individual genetic makeup.

Genetics 98

Genetics Drugs Genome Project Biobanking 98

pharmaphorum

JANUARY 18, 2023

In 2016, scientists behind a study called the Resilience Project analysed genetic data from 589,000+ people and found 13 adults who carried genetic variants that should have resulted in serious – even deadly – childhood disease, but who were apparently healthy. Giving participants something in return.

Genetic Disease 97

Genetic Disease Genetics Research Genomics 97

Pharmaceutical Technology

MARCH 2, 2023

The European Medicines Agency (EMA) is also reviewing Reata’s marketing authorisation application (MAA) for SKYCLARYS in Europe. We are grateful to Friedreich’s ataxia patients, investigators, US regulators, and our scientists and employees who made this approval possible. “As

FDA Approval 264

FDA Approval Trials Genetics Drugs 264

XTalks

MAY 20, 2022

If you enjoy working with biological samples and are enthusiastic about healthcare, a career as a clinical scientist might be just right for you. To start applying to clinical scientist jobs today, head over to the Xtalks Job Search platform. Who is a Clinical Scientist? What is it Like to Work as a Clinical Scientist?

Scientist 105

Scientist Life Science Clinical Research Doctors 105

Scienmag

MAY 7, 2021

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.

Genetics 95

Genetics Genetic Disease RNA Protein 95

Medical Xpress

NOVEMBER 22, 2022

Scientists at University of Florida (UF) Scripps Biomedical Research have developed a potential medicine for a leading cause of ALS and dementia that works by eliminating disease-causing segments of RNA. The compound restored the health of neurons in the lab and rescued mice with the disease.

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RNA Scientist Genetics Development 76

Medical Xpress

NOVEMBER 10, 2022

Scientists at the University of Pittsburgh School of Medicine have discovered the missing puzzle piece in the mystery of how melanoma tumors control their mortality.

Scientist 76

Scientist Genetics Medicine 76

Pharma Mirror

APRIL 21, 2024

With the rapid development of biotechnology and molecular medicine, the introduction of mRNA as a vaccine or therapeutic agent enables the production of almost any desired functional protein/peptide within the human body.

In-Vitro 130

In-Vitro Drugs Genetic Disease RNA 130

Medical Xpress

FEBRUARY 28, 2023

Northwestern Medicine scientists have identified the cause of a genetic subtype of autism and schizophrenia that results in social deficits and seizures in mice and humans.

Genetics 75

Genetics Scientist Medicine 75

Scienmag

MARCH 31, 2021

But now, scientists at Washington University School of Medicine in St. […].

Scientist 62

Scientist Genetics Gene Medicine 62

pharmaphorum

SEPTEMBER 14, 2020

Last week geneticist Dr Charles Steward shared with us his experiences of searching for a genetic cause for his children’s rare neurological diseases. Here he gives us a deeper look at how genomic medicine is evolving and the barriers that are preventing it from reaching its full potential.

Genome 119

Genome Genomics Medicine Gene 119

STAT News

SEPTEMBER 30, 2022

For a while, scientists thought the trillions of microbes on our bodies lived in landscapes connected to the outside world — our skin, hair, and gut — but research in the last few years has shown that’s not so. Read the rest…

Bacteria 145

Bacteria Scientist Genetics Medicine 145

STAT News

MARCH 1, 2023

Tessera Therapeutics chief scientist Michael Holmes had just finished presenting a much-anticipated peek at a technology the company had previously said could “ revolutionize genetic medicine ” and “cure nearly any genetic disease.”

Gene Editing 119

Gene Editing Genomics Genome Genetic Disease 119

Medical Xpress

JANUARY 5, 2023

Scientists at St. Jude Children's Research Hospital are reporting the most comprehensive study to date describing the variations in drug response across different genetic subtypes of acute lymphoblastic leukemia (ALL). The findings provide a blueprint for precision medicine to further individualize therapy.

Medicine 75

Medicine Genetics Scientist Drugs 75

Scienmag

JUNE 28, 2022

Whether fighting a cancer or a chronic infection, they become less effective over time, a phenomenon dubbed “T cell exhaustion” by scientists. Now, researchers at Gladstone Institutes and Stanford University have revealed the genetic switches that get […].

Scientist 80

Scientist Genetics Research Medicine 80

Scienmag

OCTOBER 13, 2021

An artificial intelligence (AI)-based technology rapidly diagnoses rare disorders in critically ill children with high accuracy, according to a report by scientists from University of Utah Health and Fabric Genomics, collaborators on a study led by Rady Children’s Hospital in San Diego.

Genetics 87

Genetics Genomics Genome Scientist 87

Scienmag

MAY 31, 2021

NIH- and USU- led study links ALS to a fat manufacturing gene and maps out a genetic therapy Credit: Courtesy of the NIH/NINDS.

Genetics 56

Genetics Scientist Gene Manufacturing 56

Medical Xpress

MAY 16, 2023

Sometimes scientists arrive at new findings and discoveries by chance. That was the case when Thilini Gamage was to carry out one of the studies in her doctoral work with Professor Eirik Frengen at the Institute of Clinical Medicine, University of Oslo. They study gene variation and mutations that cause rare genetic diseases.

In-Vitro 98

In-Vitro In-Vivo Genetic Disease Genetics 98

Scienmag

MARCH 25, 2021

Decade-long search uncovers genes responsible for lacunar strokes, a major cause of vascular dementia Scientists have identified new genetic clues in people who’ve had small and often apparently ‘silent’ strokes that are difficult to treat and a major cause of vascular dementia, according to research funded by the British Heart Foundation (..)

Genetics 75

Genetics Gene Scientist Research 75

The Pharma Data

FEBRUARY 22, 2022

State-of-the-art facility will headquarter research efforts in Boston and New York to accelerate the development of RNA and DNA-based medicines. Adams, vice president of genetic medicine at Lilly and co-director of the Institute. Adams, vice president of genetic medicine at Lilly and co-director of the Institute.

Genetics 52

Genetics Medicine RNA Scientist 52

Scienmag

JULY 7, 2021

Scientists at the University of Cambridge have identified rare genetic variants – carried by one in 3,000 people – that have a larger impact on the risk of developing type 2 diabetes than any previously identified genetic effect.

Genetics 69

Genetics Scientist Development Gene 69

Scienmag

MARCH 30, 2021

Credit: Gretel Nicholson, EXRC Scientists have discovered a new genetic disease, which causes some children’s brains to develop abnormally, resulting in delayed intellectual development and often early onset cataracts.

Genetic Disease 52

Genetic Disease Genetics Scientist Development 52

Scienmag

APRIL 8, 2021

Credit: UT Southwestern Medical Center DALLAS – April 8, 2021 – New research has uncovered a surprising role for so-called “jumping” genes that are a source of genetic mutations responsible for a number of human diseases.

Scientist 77

Scientist Gene Genetics Research 77

Pharmaceutical Technology

APRIL 20, 2023

Circio aims to develop new circRNA medicines initially for cancer, then plans to expand rapidly into vaccines and gene therapy. The circVec platform is a modular genetic cassette that provides instructions for the generation of multifunctional circRNAs.

Gene Therapy 240

Gene Therapy RNA Gene Development 240

Scienmag

JULY 6, 2021

Credit: UT Southwestern Medical Center Using artificial intelligence, UT Southwestern scientists have identified thousands of genetic mutations likely to affect the immune system in mice. The work is part of one Nobel laureate’s quest to find virtually all such variations in mammals.

Scientist 77

Scientist Genetics Gene Medicine 77

XTalks

NOVEMBER 10, 2020

Expeditious and accurate diagnoses are necessary for patients to access healthcare services and treatment options for rare genetic diseases. Increasing the efficiency of case analysis and interpretation is essential to providing timely care for patients with genetic diseases.

Genome 98

Genome Genomics Medicine Genetic Disease 98

Scienmag

NOVEMBER 22, 2021

Scientist are now combining recent advances in evolutionary analysis and deep learning to build three-dimensional models of how most proteins in eukaryotes interact.

Protein 90

Protein Scientist Genetics Medicine 90

Scienmag

NOVEMBER 11, 2020

New method bridges in situ microscopy with single cell omics Credit: Wheeler lab Scientists can now select individual cells from a population that grows on the surface of a laboratory dish and study their molecular contents.

Scientist 98

Scientist Development Research Bioinformatics 98

XTalks

OCTOBER 8, 2024

The 2024 Nobel Prize in Physiology or Medicine has been awarded to American scientists Victor Ambros and Gary Ruvkun for their groundbreaking discovery of microRNA (or miRNA) and its role in post-transcriptional gene regulation. Last year’s Nobel Prize in Physiology and Medicine also went to RNA researchers.

Regulation 52

Regulation Genetics Scientist RNA 52

Scienmag

NOVEMBER 25, 2020

Credit: Professor Jane Farrar and Dr Daniel Maloney, Trinity College Dublin Scientists from Trinity College Dublin have developed a new gene therapy approach that offers promise for one day treating an eye disease that leads to a progressive loss of vision and affects thousands of people across the globe.

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Gene Therapy Scientist Gene Development 87