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New Cages to Trap Molecules Push Boundaries of Protein Design

January 27, 2020

Protein design is a popular and rapidly growing field, with scientists engineering novel protein cages—capsule-like nanostructures for purposes…
A New Era is Dawning in Cystic Fibrosis Treatment

December 27, 2019

As her health began to worsen in late 2017, Annette Quinn started measuring her declining lung function by…
Injection of Virus-Delivered Gene Silencer Blocks ALS Degeneration, Saves Motor Function

December 24, 2019

Novel spinal therapy/delivery approach prevented disease onset in neurodegenerative ALS disease model in adult mice and blocked progression…
A new gene therapy strategy, courtesy of Mother Nature

December 17, 2019

Scientists have developed a new gene-therapy technique by transforming human cells into mass producers of tiny nano-sized particles…
Next-Gen Medicine

November 26, 2019

Harvard, MIT, teaching hospitals team up with industry partners to launch new center for regenerative therapies. From gene…
One Fell Swoop

November 7, 2019

As we age, our bodies tend to develop diseases such as heart failure, kidney failure and diabetes, and…
Penn developed Gene Therapy for Duchenne Muscular Dystrophy Safely Preserves Muscle Function

October 9, 2019

A gene therapy being developed at Penn Medicine to treat Duchenne muscular dystrophy (DMD) successfully and safely stopped the…
NIH researchers create new viral vector for improved gene therapy in sickle cell disease

October 3, 2019

Researchers at the National Institutes of Health have developed a new and improved viral vector—a virus-based vehicle that…
New CRISPR-Cas9 variant may boost precision in gene editing

October 2, 2019

Researchers have developed a new variant of the gene editing technique CRISPR-Cas9 that has the potential to increase…
Gene Therapy Update: Remembering Jesse Gelsinger

September 30, 2019

Like the mythological phoenix bird, gene therapy has risen from the ashes and is spreading its wings. September…
CWRU and Akouos sign agreement to advance development of gene therapy technology as a potential treatment for a type of Usher syndrome

September 17, 2019

Case Western Reserve University and Boston-based Akouos have entered into an exclusive licensing agreement to develop a patented gene therapy with…
Is treatment forever? Success of gene therapy for inherited blindness depends on timing

September 11, 2019

Nearly two decades ago, a gene therapy restored vision to Lancelot, a Briard dog who was born with…
Tiny capsules packed with gene-editing tools offer alternative to viral delivery of gene therapy

September 10, 2019

New tools for editing genetic code offer hope for new treatments for inherited diseases, some cancers, and even…
AstraZeneca Challenge: Improving the Targeting and Transduction Efficiency of Adeno-Associated Viral Vectors

August 21, 2019

Adeno-associated virus (AAV) is a non-enveloped virus that infects humans with no known pathogenicity. With the ability to…
Catch the Rhythm

July 22, 2019

Marked by racing, irregular heartbeats, the inherited arrhythmia known as CPVT is typically silent until a child suddenly…
When a Rare Mutation Causes a Rare Disease: Jacob’s Story

June 28, 2019

For some parents, a physician’s advice to “just take him home and love him,” presumably letting nature take…
Scientists chart course toward a new world of synthetic biology

June 20, 2019

Genetically engineered trees that provide fire-resistant lumber for homes. Modified organs that won’t be rejected. Synthetic microbes that…
Using gene editing, neuroscientists develop a new model for autism

June 19, 2019

Using the genome-editing system CRISPR, researchers at MIT and in China have engineered macaque monkeys to express a…
Replicating Fetal Bone Growth Process Could Help Heal Large Bone Defects

June 7, 2019

To treat large gaps in long bones, like the femur, which result from bone tumor removal or a…
Newly Approved Spinal Muscle Atrophy Treatment Zolgensma is Based on Delivery System Discovered by Penn Gene Therapy Pioneer

May 29, 2019

James Wilson, MD, PhD, recalls being struck by the devastating toll of rare diseases as a young physician in…
Grünenthal Challenge: Cell-Based/Gene Therapies for Pain

May 29, 2019

Pain is an unpleasant sensation that is part of the body’s protective mechanism designed to give a warning…
Researchers Use Gene Editing with CRISPR to Treat Lethal Lung Diseases Before Birth

April 19, 2019

Using CRISPR gene editing, a team from Penn Medicine and Children’s Hospital of Philadelphia (CHOP) have thwarted a lethal lung disease, in an…
Gene therapy restores immunity in infants with rare immunodeficiency disease

April 19, 2019

A small clinical trial has shown that gene therapy can safely correct the immune systems of infants newly…
Scientists studying Krabbe’s disease take a novel approach to this fatal disease that strikes newborns

April 2, 2019

NIH has awarded $2 million to UB’s Hunter James Kelly Research Institute to pursue a new direction in…
Elderly couple. Image credit: Bridget Coila
Gene Therapy Shows Initial Promise for Parkinson’s Disease

March 22, 2019

A delicate operation that involved placing a gene into the brain was found to reduce the severity of…
With single gene insertion, blind mice regain sight

March 20, 2019

It was surprisingly simple. University of California, Berkeley, scientists inserted a gene for a green-light receptor into the…
Scientists found a way to use gene therapy to combat Chlamydia

February 13, 2019

Chlamydia is the most common sexually transmitted bacterial infection in the world. With a growing threat of antibiotic-resistant…
Gene Therapy Treatment Targets Rare Mutation Tied to Blindness

February 13, 2019

Eye troubles detected at infancy began to take their toll when Emma Kate Roden started walking. The toddler…
Initiative Will Create Coursework for Cell Manufacturing Workers

January 29, 2019

An 18-month federally-sponsored project led by the Georgia Institute of Technology will develop much-needed curriculum to train workers…
Gene therapy blocks peripheral nerve damage in mice

January 22, 2019

Nerve axons serve as the wiring of the nervous system, sending electrical signals that control movement and sense…
Engineers create an inhalable form of messenger RNA

January 7, 2019

Messenger RNA, which can induce cells to produce therapeutic proteins, holds great promise for treating a variety of…
Injection Improves Vision in a Form of Childhood Blindness

December 19, 2018

A new treatment for patients with a form of congenital retinal blindness has shown success in improving vision,…
New understanding of mysterious ‘hereditary swelling’

December 13, 2018

An upper arm that swells to twice its normal size. A stomach that inexplicably swells up causing colic…
The First Genome Surgeons: Scientists Are Preparing to Bring DNA-Editing Tools to the Clinic

October 30, 2018

One afternoon in July, deep within the labyrinthine halls of the Medical Sciences Building at UC San Francisco’s…
CRISPR opens door to new type of medicine: ‘genome surgery’

October 29, 2018

Within a few years, Jim Johnsen and Delaney Van Riper may be among the first to benefit from…
Gene Plays Critical Role in Noise-Induced Deafness

October 24, 2018

In experiments using mice, a team of UC San Francisco researchers has discovered a gene that plays an…
This RNA-based technique could make gene therapy more effective

October 17, 2018

Delivering functional genes into cells to replace mutated genes, an approach known as gene therapy, holds potential for…
Gene therapy breakthrough in treating rare form of blindness

October 10, 2018

Positive results of the world’s first gene therapy trial for a genetic cause of blindness known as choroideremia…
Red Glow Helps Identify Nanoparticles for Delivering RNA Therapies

October 4, 2018

A new screening process could dramatically accelerate the identification of nanoparticles suitable for delivering therapeutic RNA into living…
Gene therapy for mitochondrial diseases

October 2, 2018

Mitochondrial disease is now thought to be the second most commonly diagnosed genetic disease worldwide, and, unfortunately, there…
Mitochondrial diseases could be treated with gene therapy, study suggests

September 25, 2018

Researchers have developed a genome-editing tool for the potential treatment of mitochondrial diseases: serious and often fatal conditions…
Researchers find potential new gene therapy for blinding disease

August 24, 2018

Scientists funded by the National Eye Institute (NEI) report a novel gene therapy that halts vision loss in…
Knockdown and replace: A gene therapy twofer to treat blindness

August 22, 2018

The last year has seen milestones in the gene therapy field, with FDA approvals to treat cancer and…
Targeted Gene Editing Cures Blood Disorder in Fetal Mice

July 30, 2018

A team of researchers, including UConn assistant professor of pharmaceutics Raman Bahal, has, for the first time, corrected a…
Novel drug therapy partially restores hearing in mice

July 10, 2018

A small-molecule drug is one of the first to preserve hearing in a mouse model of an inherited…
Gene therapy method developed to target damaged kidney cells

July 10, 2018

Gene therapy has gained momentum in the past year, following the federal government’s approval of the first such…
Study points researchers toward new therapies for fragile X syndrome

July 9, 2018

New insights into the molecular machinations behind fragile X syndrome, the most common inherited intellectual disability, may help…
Correcting a blind spot

July 2, 2018

The human eye is a wonder. It is often said to be the second most complex organ in…
Potential treatment for genetic form of autism

June 29, 2018

A team of researchers, led by Professor Riccardo Brambilla from Cardiff University, discovered that an experimental drug can…
With gene editing, researchers cure blood disorder in fetal mice

June 27, 2018

With the combined efforts of three Yale laboratories, researchers conducted the first demonstration of site-specific gene editing in…
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