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Gene therapy challenge: How much should it cost and how do we pay for it?

When evaluating the high prices of one-time gene therapy treatments, should we look to the costs of organ transplants when ...
Fighting infectious diseases with immunotherapy on 'cusp of commercialization'

Fighting infectious diseases with immunotherapy on ‘cusp of commercialization’

Immunotherapy, which involves adapting immune cells to destroy specific cellular targets, has made a name for itself treating cancer. But ...
Hemophilia 12/7/17

Gene therapy could be ‘ideal cure’ for hemophilia B

[L]ast year, [Jay] Konduros enrolled in a clinical trial, receiving an experimental gene therapy at Children's Hospital of Philadelphia [to ...
DIY gene therapy? FDA is not a fan

DIY gene therapy? FDA is not a fan

A few biohackers (fans of do-it-yourself (DIY) science experiments) recently designed a gene therapy for HIV. Not only that, they posted a video ...
Exorbitant costs of gene therapy raise payment concerns for patients and government

Exorbitant costs of gene therapy raise payment concerns for patients and government

[Editor's note: Executives from several companies developing gene therapies gathered for a panel discussion at the recent Forbes Healthcare Summit, ...
Genetic engineering, synthetic biology poised to boost photosynthesis and carbon capture

Genetic engineering, synthetic biology poised to boost photosynthesis and carbon capture

In a feature article published in the open access journal eLife, an international team of experts led by Dr Bonnie Wintle ...
Insecticide resistance threatens to derail malaria control in Africa

Insecticide resistance threatens to derail malaria control in Africa

The largest genetic study of mosquitoes has found their ability to resist insecticides is evolving rapidly and spreading across Africa, ...
Can we solve the patent issues that threaten CRISPR research?

Can we solve the patent issues that threaten CRISPR research?

[Editor's note: Lawrence Horn is President and CEO of MPEG LA, which provides licenses for standards and other technology platforms.] Although recent ...
Why life-saving gene therapy isn't available yet to children who need it most

Why life-saving gene therapy isn’t available yet to children who need it most

[The first patient to permanently edit his DNA,] Brian Madeux, 44, of Arizona, is part of a clinical trial testing ...
Gene therapy challenge: Overcoming shortage of key and expensive viruses

Gene therapy challenge: Overcoming shortage of key and expensive viruses

Eager to speed development of revolutionary treatments, the Food and Drug Administration recently announced that it would expedite approval of ...
How gene therapy could help fight methamphetamine addiction

How gene therapy could help fight methamphetamine addiction

Gene therapy, which modifies a person’s DNA, has long been thought of as a way to treat genetic diseases—and, more recently, ...
Exploring national security risks related to gene editing

Exploring national security risks related to gene editing

Technology is advancing exponentially and the exciting field of genome editing is no exception. Scientists at the University of Wisconsin-Madison ...
Viewpoint: Cost of gene therapy could put experimental treatments out of reach for most

Viewpoint: Cost of gene therapy could put experimental treatments out of reach for most

Drugs that modify human genes have the potential to cure intractable diseases with just one treatment. Few could disagree that's ...
Canadian researchers can't use CRISPR in human embryo research

Canadian researchers can’t use CRISPR in human embryo research

In the United States, using genetic engineering techniques such as CRISPR to make genetic alterations that can be passed on ...
FDA details risk-based cellular therapy and regenerative medicine guidelines

FDA details risk-based cellular therapy and regenerative medicine guidelines

The FDA has issued two final guidances and two draft guidances, all designed to articulate the agency’s approach to developing ...
Talking Biotech: Pet dogs with genetic diseases testing ground for gene therapy

Talking Biotech: Pet dogs with genetic diseases testing ground for gene therapy

Journalist Emily Mullin: Gene therapies becoming more promising but restricted in humans because of safety concerns, prompting some researchers to ...
New T-cell treatment shows promise for treating leukemia

New T-cell treatment shows promise for treating leukemia

A new way of genetically altering a patient’s cells to fight cancer has helped desperately ill people with leukemia when ...
Gene therapy boost: FDA positions for faster reviews of new treatments

Gene therapy boost: FDA positions for faster reviews of new treatments

The Food and Drug Administration on [November 16] issued new guidelines to speed the introduction of treatments involving human cells ...
FDA likely to approve hereditary blindness gene therapy

FDA likely to approve hereditary blindness gene therapy

[A]nother gene therapy is on the cusp of approval, this time to treat a form of hereditary blindness. If given ...
A gene-editing first: Scientists try to edit a living human's DNA

A gene-editing first: Scientists try to edit a living human’s DNA

Scientists for the first time have tried editing a gene inside the body in a bold attempt to permanently change ...
Ethical debates swirl around gene-editing decisions

Ethical debates swirl around gene-editing decisions

[Editor's note: Joselin Linder is the author of the book "The Family Gene: A Mission to Turn My Deadly Inheritance ...
New error-free DNA sequencing method could diagnose rare diseases

New error-free DNA sequencing method could diagnose rare diseases

A virtually error-free new method of DNA sequencing could one day be used to diagnose extremely rare cancers and hereditary ...
The quest to make CRISPR gene editing as easy as a smartphone app

The quest to make CRISPR gene editing as easy as a smartphone app

[Biohacker Josiah Zayner] lives and works in Oakland, California, where he's converted a house into a scientific lab. Here, he ...
Gene therapy creates boy's replacement skin from his stem cells

Gene therapy creates boy’s replacement skin from his stem cells

Doctors treated a 7-year-old boy’s devastating genetic skin disease—junctional epidermolysis bullosa—by genetically modifying his stem cells to create a new ...
What's the excitement over gene therapy?

What’s the excitement over gene therapy?

Gene therapy has been in the news a lot of late as a potential cure for a variety of genetic ...
Safer CRISPR? Cas13 version edits RNA, not DNA

Safer CRISPR? Cas13 version edits RNA, not DNA

CRISPR, while a major leap forward in gene editing, can still be a blunt instrument. There have been problems with ...
Fighting malaria: Bacteria targets parasites in mosquito gut

Fighting malaria: Bacteria targets parasites in mosquito gut

Using a bacteria strain that colonizes the mosquito digestive tract and spreads rapidly throughout the populations, scientists have successfully inhibited ...
Biohackers help 'patient' inject himself with experimental HIV treatment—live on Facebook

Biohackers help ‘patient’ inject himself with experimental HIV treatment—live on Facebook

[Tristan] Roberts is about to inject himself with an experimental gene therapy for HIV, a DIY prototype treatment designed by ...