Advanced Search
Current and Breaking News for Professionals, Consumers and Media

Click here to learn how to advertise on this site and for ad rates.

Genetic Issues Author: Staff Editor Last Updated: Sep 7, 2017 - 10:06:33 PM

New Way of Genome Editing Could Cure Hemophilia in Mice

By Staff Editor
Oct 30, 2014 - 1:17:59 PM

Email Newsletter icon, E-mail Newsletter icon, Email List icon, E-mail List icon Sign up for our Ezine
For Email Marketing you can trust

Email this article
 Printer friendly page

( - A technique developed by Stanford researchers could provide a safer, longer-lasting method of replacing faulty, disease-causing genes with working copies.

The ability to pop a working copy of a faulty gene into a patient's genome is a tantalizing goal for many clinicians treating genetic diseases. Now, researchers at the Stanford University School of Medicine have devised a new way to carry out this genetic sleight of hand.

The approach differs from that of other hailed techniques because it doesn't require the co-delivery of an enzyme called an endonuclease to clip the recipient's DNA at specific locations. It also doesn't rely on the co-insertion of genetic "on" switches called promoters to activate the new gene's expression.

These differences may make the new approach both safer and longer-lasting. Using the technique, the Stanford researchers were able to cure mice with hemophilia by inserting a gene for a clotting factor missing in the animals.

"It appears that we may be able to achieve lifelong expression of the inserted gene, which is particularly important when treating genetic diseases like hemophilia and severe combined immunodeficiency," said Mark Kay, MD, PhD, professor of pediatrics and of genetics. "We're able to do this without using promoters or nucleases, which significantly reduces the chances of cancers that can result if the new gene inserts itself at random places in the genome."

Using the technique, Kay and his colleagues were able to insert a working copy of a missing blood-clotting factor into the DNA of mice with hemophilia. Although the insertion was accomplished in only about 1 percent of liver cells, those cells made enough of the missing clotting factor to ameliorate the disorder.

Kay is the senior author of the research, published Oct. 29 in Nature. The lead author is postdoctoral scholar Adi Barzel, PhD.


For advertising/promos call Mike McCurdy at 877-634-9180 or email at [email protected] We have over 7,000 journalists who are subscribers.

Top of Page

Genetic Issues
Latest Headlines

+ Link a Relatively Unknown Gene to Early Emergence of Blood Diseases
+ How Gene Mutation Triggers Immune Disease
+ Giving Silenced Genes a Voice
+ A Centuries-Old Math Equation Used to Solve a Modern-Day Genetics Challenge
+ Genetic ‘Switches’, Mapped for First Time, Drive Human Brain Development
+ St. Jude Gene Therapy Improves Immunity in Babies with ‘Bubble Boy’ Disease
+ Researchers Find Genes May ‘Snowball’ Obesity
+ Number of Genetic Markers Linked to Lifespan Triples
+ Do Your Ears Hang Low? The Complex Genetics Behind Earlobe Attachment
+ Genetic Predisposition to Later Puberty Causes Lower Bone Density in Children and Adults

Contact Us | Job Listings | Help | Site Map | About Us
Advertising Information | HND Press Release | Submit Information | Disclaimer

Site hosted by Sanchez Productions