Gene Therapy

Ageing is a disease. Gene therapy could be the 'cure'

After her son was diagnosed with type 1 diabetes, Elizabeth Parrish began researching gene therapy treatments. She then started testing them on herself<p>In September 2015, Elizabeth Parrish flew from Seattle to Colombia to receive an experimental treatment.<p>Craig Venter wants to tackle the ultimate …

Genetics

Artificial Intelligence takes Gene Therapy to the next level

<b>Medicine</b> remains one of the most challenging fields for scientists to solve problems, but a new technique to combat rare diseases is becoming …

Genetics

Regenxbio Sinks After Announcing Secondary Offering

Regenxbio Inc. (NASDAQ: RGNX) saw its shares drop handily after the firm announced the pricing of its secondary offering. The company plans to offer …

Genetics

Buy Regenxbio Shares On Current Weakness

Shares are up around 50% since my initial recommendation.<p>Current weakness is a buying opportunity.<p>Cash position provides a runway for 2.5 years give …

Stocks

Determined Parents are Moving the Needle on Gene Therapy

Families of patients are starting advocacy groups, raising money for research, and founding biotech companies to advance cures for rare …

Genetics

Gene therapy hope for blindness

John von RadowitzPress Association<p><b>A gene therapy treatment for the most common cause of blindness in young people is being tested in a group of</b> …

Genetics

Gene therapy successfully cures two babies of cancer

WHY THIS MATTERS IN BRIEF<p>New gene therapy treatments could spell the end of painful and exhausting chemotherapy treatments, and give patients new …

Cancer

Top 10 'breakthrough' technologies for 2017

The technologies making waves in 2017 include brain implants and quantum computers.<p>Here is a list of the top 10 technologies that are expected to be prevalent this year, according to MIT.<p><b>AI that learns like humans</b><p>At the top of the list is behavior-reinforced artificial intelligence.<p>Whether that’s …

Genetics

Rare Bone Marrow Transplant Saves Eight-Month-old Afghan Baby

Combined Immunodeficiency is in which a person is born without a functioning immune system, increasing his or her risk of developing fatal infections.

Medicine

Terminal Cancer Patients Lives to See Another Day With Thanks to Revolutionary Gene Therapy

A new and exciting type of gene therapy treatment has been implemented as so far its results have been incredible. It works by altering the patient’s …

Cancer

“In the Coming Decade, Off-the-Shelf Cell Therapy will Become a Reality”

<b>David Sourdive, co-founder of Cellectis, discusses his pioneering work developing off-the-shelf CAR-T for cancer and the future directions of cell</b> …

Innovation

Living With Sickle Cell: ‘I Don’t Know What It Means to Be Without Pain’

Nikki Peterson, like approximately 100,000 other Americans, was born with sickle cell anemia. The 43-year-old lives in Upper Marlboro, Md., and ends …

Medicine

A cure for millions as gene therapy cures patient with Sickle Cell disease

WHY THIS MATTERS IN BRIEF<p>Sickle cell disease is a painful and debilitating disease that affects millions of people worldwide, but everything seems to …

Medicine

Tocagen Plans IPO to Finance Trials of Gene Therapy in Brain Cancer

[<i>Corrected 3/10/17, 3:27 pm. See below</i>] Gene therapy developer Tocagen is preparing an initial public stock offering to finance clinical trials of …

Cancer

Pfizer looks at building major gene therapy manufacturing facility in North Carolina

Pfizer, which scooped up Bamboo Therapeutics last year in its aim to be a major player in gene therapies, is now looking at building a gene therapy …

Genetics

This Lab-in-a-Box Could Make Gene Therapy Less Elitist

Genetic repairs are curing patients—but only at a few elite centers.<p>Jennifer Adair carried out her first gene therapy experiment several years ago. A …

Genetics

Gene Therapy Needs Machines

News came recently of an apparent cure, via gene therapy, of sickle-cell disease in a young patient (whose condition was refractory to hydroxyurea …

Genetics

The Download, Mar 8, 2017: CIA Leak Realism, Democratizing Gene Therapy, and Luxurious Smartphones

The most fascinating and important news in technology and innovation delivered straight to your inbox, every day.<p>Three Things You Need to Know</b> …

Genetics

Gene Therapy in a New Light

The small, windowless space at the Children's Hospital of Philadelphia looks like any eye doctor's examining room, with an adjustable chair and half …

Genetics

Dosing underway in REGENXBIO's early-stage study of gene therapy RGX-501 in HoFH

The first patient has been dosed in REGENXBIO's (NASDAQ:RGNX) Phase 1/2 clinical trial assessing gene therapy RGX-501 for the treatment of homozygous …

Genetics

In a first, gene therapy lets teen dodge sickle cell disease

Teen who was given gene therapy for sickle cell disease now has enough properly working red blood cells to dodge the effects of the disorder, …

Medicine

Risks - Gene therapy

Gene therapy has some potential risks. A gene can't easily be inserted directly into your cells. Rather, it usually has to be delivered using a …

Genetics

Scientists Reverse Sickle Cell Disease for the First Time Using Gene Therapy

This is so cool.<p>A new gene therapy technique has been used to successfully reverse sickle cell disease for the first time, scientists report.<p>While …

Genetics

Sickle cell anemia patient 'cured' by gene therapy, doctors say

<b>(CNN) —</b> In a world first, a teenager with sickle cell disease achieved complete remission after an experimental gene therapy at Necker Children's Hospital in Paris, researchers say.<p>People with sickle-cell disease, a group of inherited blood disorders, have abnormal hemoglobin in their red blood …

Medicine

Doctors reverse teen's sickle cell disease with innovative gene therapy

A French teen who underwent a first-of-its-kind procedure 15 months ago to change his DNA shows no signs of the sickle cell disease he had been suffering from. The procedure, which was performed at Necker Children’s Hospital in Paris, may offer hope to millions of patients who suffer from sickle …

Medicine

Bluebird Bio’s New Treatment Cured Sickle Cell in a Teenage Boy

A promising new sickle cell gene therapy procedure has left a French teenager with no sign of the disease 15 months after treatment, raising hopes …

Medicine

Gene therapy relieves sickle cell in world first: study

<b>Scientists have used gene therapy to relieve the symptoms of a teenager suffering from sickle cell disease (SCD) in a world-first breakthrough, they</b> …

Genetics

Gene therapy shows early promise against sickle cell

Researchers are reporting early success using gene therapy to treat, or even potentially cure, sickle cell anemia.<p>The findings come from just one patient, a teenage boy in France. But more than 15 months after receiving the treatment, he remained free of symptoms and his usual medications.<p>That's a …

Genetics

10 Breakthrough Technologies 2017

Reversing Paralysis<p>Scientists are making remarkable progress at using brain implants to restore the freedom of movement that spinal cord injuries …

Genetics

Sickle cell gene therapy: 'It gives me joy and hope'

<b>Sia Evelyn Nyandemo, originally from Sierra Leone, runs a campaign group to support sickle cell sufferers. She spoke to the BBC's Newsday programme about caring for her daughter who has the disease and her reaction to a new treatment which could offer a possible cure to the life-threatening</b> …

Genetics