Upcoming Webinars Archived Webinars Training Vitals Host A Webinar About Get Updates Contact

New CRISPR Gene Editing Enzyme Removed Duchenne Muscular Dystrophy Mutations

XTALKS VITALS NEWS

DNA

While the researchers previously used the original CRISPR-Cas9 system, they have now used a new enzyme, CRISPR-Cpf1, to change the defect in both mouse and human cells.

Share this!

April 17, 2017 | by Sarah Hand, M.Sc.

For the second time, a team of researchers at UT Southwestern Medical Center have corrected mutations associated with Duchenne muscular dystrophy using CRISPR gene editing. While the researchers previously used the original CRISPR-Cas9 system, they have now used a new enzyme, CRISPR-Cpf1, to change the defect in both mouse and human cells.

“We took patient-derived cells that had the most common mutation responsible for Duchenne muscular dystrophy and we corrected them in vitro to restore production of the missing dystrophin protein in the cells. This work provides us with a promising new tool in the CRISPR toolbox,” said senior study author, Dr. Eric Olson, Chairman of Molecular Biology, Co-Director of the UT Southwestern Wellstone Muscular Dystrophy Cooperative Research Center, and Director of the Hamon Center for Regenerative Science and Medicine. The researchers published their findings in the journal, Science Advances.

The CRISPR-Cpf1 tool has a number of advantages over the original CRISPR-Cas9 gene editing system. Importantly, the Cpf1 enzyme is smaller than Cas9, which allows it to more easily fit inside of a viral vector which can deliver the enzyme to muscle cells. Cpf1 also recognizes a different sequence, giving researchers options when it comes to gene editing.

“There will be some genes that may be difficult to edit with Cas9 but may be easier to modify with Cpf1, or vice versa,” said Olson. “The two proteins have different biochemical properties and recognize different DNA sequences, so these properties create more options for gene-editing.”



Mutations in the dystrophin gene – one of the longest genes ever identified – cause Duchenne muscular dystrophy. Dystrophin proteins provide a supportive role for muscle fiber, and its absence causes a number of characteristic symptoms, including progressive muscle weakness and eventually loss of muscle function.

“By either skipping a mutation region or precisely repairing a mutation in the gene,” said co-author Dr. Rhonda Bassel-Duby, Professor of Molecular Biology and Associate Director of the Hamon Center for Regenerative Science and Medicine, “CRISPR-Cpf1-mediated genome editing not only corrects Duchenne muscular dystrophy mutations but also improves muscle contractility and strength.”

Because of concerns surrounding so-called “off-target” effects of CRISPR gene editing, the researchers must find a way to ensure that the technique is focused and controlled. “CRISPR-Cpf1 gene-editing can be applied to a vast number of mutations in the dystrophin gene. Our goal is to permanently correct the underlying genetic causes of this terrible disease, and this research brings us closer to realizing that end,” said Olsen.

According to the Centers for Disease Control and Prevention (CDC), Duchenne muscular dystrophy occurs with a frequency of one in every 5,000 boys. The FDA recently approved two drugs, Sarepta Therapeutics’ Exondys 51 and Marathon Pharmaceuticals’ Emflaza, to treat Duchenne muscular dystrophy.


Keywords:  CRISPR, Duchenne Muscular Dystrophy, Gene Editing


| NEXT ARTICLE | MORE NEWS | BLOGS | VIDEOS | POLLS & QUIZZES | WEBINARS |

Share this with your colleagues!

MORE NEWS
Exclusion Criteria for Clinical Trials Poses Major Barrier to Patient Enrollment

August 17, 2017 - UT Southwestern researchers say that clinical investigators continue to increase the number of exclusion criteria, preventing more patients from participating in clinical trials.

Featured In: Clinical Trials News


Targeting Cellular Nitrogen Metabolism Could Offer a New Treatment for Pancreatic Cancer

August 17, 2017 - An enzyme involved in regulating the amount of nitrogen in the cell could be a new drug target for pancreatic cancer, according to researchers from Boston Children's Hospital and the Broad Institute of MIT and Harvard.

Featured In: Life Science News


Regeneron’s Drug for Respiratory Syncytial Virus Fails in Phase III Clinical Trial

August 16, 2017 - Biotechnology company Regeneron has announced it will not continue development of its antibody drug, suptavumab, after a failure in a Phase III clinical trial.

Featured In: Clinical Trials News


LEAVE A COMMENT
 
  
THE XTALKS VITALS INDUSTRY BLOG

One Patient’s Perspective on Clinical Trials

REGISTER FOR THESE WEBINARS

Planning and Conducting Trials of the Latest Immunotherapies


ISO 13485:2016 for Medical Device Manufacturers: Ensuring a Smooth Transition through Effective Preparation


Medical Devices: Reviewing Regulatory Changes in the US and EU


Moving Beyond Regulatory and Performance Metrics in Starting Clinical Trials


Copyright © 2016-2017 Honeycomb Worldwide Inc.