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Orchard Therapeutics opens new facility for technical operations in San Francisco Bay area

Orchard Therapeutics, a clinical-stage biotechnology company dedicated to transforming the lives of patients with rare disorders through innovative gene therapies, today announced the opening of a second facility for technical operations in the San Francisco Bay Area.

1 Dec 2017

New targeted treatment could benefit patients with certain pancreatic tumors

Researchers at the University of Cincinnati College of Medicine have shown that a new targeted treatment could benefit patients with certain pancreatic tumors by preventing spread of the cancer and protecting their heart from damage--a direct result of the tumor.

1 Dec 2017

Researchers create first semi-synthetic organism that can store and use unnatural genetic information

Researchers from The Scripps Research Institute (TSRI) have designed a bacterium that incorporates two unnatural bases, called X and Y, into genes, in addition to the four natural DNA bases − G, C, A and T − that usually code for proteins.

30 Nov 2017

Study focuses on improving pharmacokinetic properties of therapeutic aptamers

Designing new therapeutic DNA aptamers with diverse side chains can improve their ability to interact with targets, and a new study describes characteristics of these side chains that may determine how long the aptamers remain in the bloodstream.

30 Nov 2017

UT Southwestern researchers develop improved gene-editing technique to correct DMD mutations

Regenerative medicine researchers at UT Southwestern Medical Center developed an improved and simplified gene-editing technique using CRISPR/Cas9 tools to correct a common mutation that causes Duchenne muscular dystrophy.

30 Nov 2017

Researchers discover critical trigger for leading form of vision loss

In a major step forward in the battle against macular degeneration, the leading cause of vision loss among the elderly, researchers at the University of Virginia School of Medicine have discovered a critical trigger for the damaging inflammation that ultimately robs millions of their sight.

27 Nov 2017

Research findings offer prime drug targets to treat rare liver cancer that strikes young people

After years of rigorous research, a team of scientists has identified the genetic engine that drives a rare form of liver cancer. The findings offer prime targets for drugs to treat the usually lethal disease, fibrolamellar hepatocellular carcinoma (FL-HCC), which mainly strikes adolescents and young adults.

22 Nov 2017

Researchers uncover how rogue immune cells gain access to neurons in multiple sclerosis

Drug designers working on therapeutics against multiple sclerosis should focus on blocking two distinct ways rogue immune cells attack healthy neurons, according to a new study in the journal Cell Reports.

21 Nov 2017

Scientists develop novel technology that uses CRISPR to map genetic mutations

A team of scientists led by Virginia Commonwealth University physicist Jason Reed, Ph.D., have developed new nanomapping technology that could transform the way disease-causing genetic mutations are diagnosed and discovered.

21 Nov 2017

Review examines effectiveness of lipid-based products to treat dry eye disease

New treatments for dry eye disease that deliver lipids to the ocular surface are designed to more closely mimic the important tear film lipid layer at the air-water interface in the eye.

From Santen Pharmaceutical Co., Ltd. 21 Nov 2017

$10 million gift creates new center at Northwestern University to study effects of environment on genes

A new $10 million gift from University trustees and supporters Louis A. Simpson '58 and Kimberly K. Querrey will create a center at Northwestern University Feinberg School of Medicine to study the effects of environment on the activation and expression of genes.

21 Nov 2017

Gene editing within a living person for the first time

Genetic editing has been performed in the laboratory until now. Living cells within Petri-dishes have been put under the microscope while gene editing tools such as CRISPR were used to cut, nick and replace parts of the DNA or genome that is defective with bits of DNA that is correct in its sequence. Now doctors in California have attempted to edit cells within a living person.

19 Nov 2017

Hibernating ground squirrels offer clues to fight against stroke-induced brain damage

In the fight against brain damage caused by stroke, researchers have turned to an unlikely source of inspiration: hibernating ground squirrels.

17 Nov 2017

Old World monkeys could hold key to stop progression of rheumatoid arthritis

In the quest for a new and more effective treatment for rheumatoid arthritis, researchers from the Keck School of Medicine of USC looked to a primate that mostly roams the land in Asia, the Middle East and Africa.

17 Nov 2017

Experts identify mental exercise program that can reduce risk of dementia

Aging research specialists have identified, for the first time, a form of mental exercise that can reduce the risk of dementia.

17 Nov 2017

Stem cell therapy fails to improve walking ability in people with PAD

A stem cell therapy did not improve walking ability in people with peripheral artery disease, although exercise did lead to significant improvements, according to the results of a new Northwestern Medicine clinical trial.

15 Nov 2017

Clinical trial results could lead to treatment of heart disease in Duchenne muscular dystrophy patients

After boys and young men with Duchenne muscular dystrophy received cardiac progenitor cell infusions, medical tests indicated that the patients' hearts appeared improved, results from a new study show. Patients in the study also scored higher on arm strength tests after receiving the cell infusions.

15 Nov 2017

Nanowires could be potential drug delivery tools for neurodegenerative diseases

Millions of Americans suffer from neurodegenerative diseases such as Alzheimer's and Parkinson's. Researchers have identified promising new treatments, such as cerebrolysin, but current clinical approaches are ineffective because critical concentrations of the drug dissipate within the body before reaching the blood-brain barrier and central nervous system.

15 Nov 2017

Tulane professor wins Department of Defense grant for research into arteriovenous malformations

Tulane University's Stryder Meadows, a cell and molecular biology professor, received a $1.7 million grant from the Department of Defense to study how arteriovenous malformations (AVM), which are defects in arteries, veins and capillaries, form Hereditary Hemorrhagic Telangiectasia (HHT), a genetic disorder that affects about 1 in 5,000 people.

14 Nov 2017

Breakthrough discovery could radically change approach to treating Klebsiella infections

Researchers at Queen's University Belfast together with the University of Vienna have discovered that treatment for the antibiotic-resistant bacteria Klebsiella pneumoniae could lie within our bodies' natural defenses.

14 Nov 2017

Therapeutics News and Research

Orchard Therapeutics opens new facility for technical operations in San Francisco Bay area

New targeted treatment could benefit patients with certain pancreatic tumors

Researchers create first semi-synthetic organism that can store and use unnatural genetic information

Study focuses on improving pharmacokinetic properties of therapeutic aptamers

UT Southwestern researchers develop improved gene-editing technique to correct DMD mutations

Researchers discover critical trigger for leading form of vision loss

Research findings offer prime drug targets to treat rare liver cancer that strikes young people

Researchers uncover how rogue immune cells gain access to neurons in multiple sclerosis

Scientists develop novel technology that uses CRISPR to map genetic mutations

Review examines effectiveness of lipid-based products to treat dry eye disease

$10 million gift creates new center at Northwestern University to study effects of environment on genes

Gene editing within a living person for the first time

Hibernating ground squirrels offer clues to fight against stroke-induced brain damage

Old World monkeys could hold key to stop progression of rheumatoid arthritis

Experts identify mental exercise program that can reduce risk of dementia

Stem cell therapy fails to improve walking ability in people with PAD

Clinical trial results could lead to treatment of heart disease in Duchenne muscular dystrophy patients

Nanowires could be potential drug delivery tools for neurodegenerative diseases

Tulane professor wins Department of Defense grant for research into arteriovenous malformations

Breakthrough discovery could radically change approach to treating Klebsiella infections

Industry Focus eBook - Cell biology (5th edition) eBook

Bringing real-time analytics to downstream bioprocess monitoring

Designing fluidic architecture for scalable diagnostic platforms

Evaluating new approaches for rare disease genomics

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