Firefly Gene Illuminates Ability of Optimized CRISPR-Cpf1 to Efficiently Edit Human Genome JUPITER, FL June 19, 2017 Scientists on the Florida campus of The Scripps Research Institute (TSRI) have improved a state-of-the-art gene-editing technology to advance the system's ability to target, cut and paste genes within human and animal cells and broadening the ways the CRISPR-Cpf1 editing system may be used to study and fight human diseases.
Professor Michael Farzan, co-chair of TSRIs Department of Immunology and Microbiology, and TSRI Research Associate Guocai Zhong improved the efficiency of the CRISPR-Cpf1 gene editing system by incorporating guide RNAs with multiplexing capability. Guide RNAs are short nucleic acid strings that lead the CRISPR molecular scissors to their intended gene targets. The TSRI discovery means multiple genetic targets in a cell may be hit by each CRISPR-Cpf1 complex.
This system simplifies and significantly improves the efficiency of simultaneous editing of multiple genes, or multiple sites of a single gene, Zhong said. This could be very useful when multiple disease-related genes or multiple sites of a disease-related gene need to be targeted.
This approach improves gene editing for a number of applications, Farzan added. The system makes some applications more efficient and other applications possible.
This study was published as an advanced online paper in the journal Nature Chemical Biology on June 19, 2017.
TSRI Advance Makes CRISPR More Efficient
Short for Clustered Regularly Interspaced Short Palindromic Repeat, the CRISPR gene editing system exploits an ancient bacterial immune defense process. Some microbes thwart viral infection by sequestering a piece of a virus foreign genetic material within its own DNA, to serve as a template. The next time the viral sequence is encountered by the microbe, its recognized immediately and cut up for disposal with the help of two types of RNA. Molecules called guide RNAs provide the map to the invader, and CRISPR effector proteins act as the scissors that cut it apart.
Over the last five years, the CRISPR gene editing system has revolutionized microbiology and renewed hopes that genetic engineering might eventually become a useful treatment for disease. But time has revealed the technologys limitations. For one, gene therapy currently requires using a viral shell to serve as the delivery package for the therapeutic genetic material. The CRISPR molecule is simply too large to fit with multiple guide RNAs into the most popular and useful viral packaging system.
The new study from Farzan and colleagues helps solve this problem by letting scientists package multiple guide RNAs.
This advance could be important if gene therapy is to treat diseases such as hepatitis B, Farzan said. After infection, hepatitis B DNA sits in liver cells, slowly directing the production of new viruses, ultimately leading to liver damage, cirrhosis and even cancer. The improved CRISPR-Cpf1 system, with its ability to multiplex,' could more efficiently digest the viral DNA, before the liver is irrevocably damaged, he said.
Efficiency is important. If you modify 25 cells in the liver, it is meaningless. But if you modify half the cells in the liver, that is powerful, Farzan said. There are other good cases say muscular dystrophy where if you can repair the gene in enough muscle cells, you can restore the muscle function.
Two types of these molecular scissors are now being widely used for gene editing purposes: Cas9 and Cpf1. Farzan said he focused on Cpf1 because it is more precise in mammalian cells. The Cpf1 molecule they studied was sourced from two types of bacteria, Lachnospiraceae bacterium and Acidaminococus sp., whose activity has been previously studied in E. coli. A key property of these molecules is they are able to grab their guide RNAs out of a long string of such RNA; but it was not clear that it would work with RNA produced from mammalian cells. Guocai tested this idea by editing a firefly bioluminescence gene into the cell's chromosome. The modified CRISPR-Cpf1 system worked as anticipated.
This means we can use simpler delivery systems for directing the CRISPR effector protein plus guide RNAs, Farzan said. Its going to make the CRISPR process more efficient for a variety of applications.
Looking forward, Farzan said the Cpf1 protein needs to be more broadly understood so that its utility in delivering gene therapy vectors can be further expanded.
In addition to Farzan and Zhong, the authors of the study, Cpf1 proteins excise CRISPR RNAs from mRNA transcripts in mammalian cells, included Haimin Wang, and Mai H. Tran of TSRI; and Yujun Li of TSRI and Wu Lien-Teh Institute, Harbin Medical University, Harbin, China.
The study was supported by the National Institutes of Health (grants R37 AI091476 and P01 AI100263).
About The Scripps Research Institute
The Scripps Research Institute (TSRI) is one of the worlds largest independent, not-for-profit organizations focusing on research in the biomedical sciences. TSRI is internationally recognized for its contributions to science and health, including its role in laying the foundation for new treatments for cancer, rheumatoid arthritis, hemophilia, and other diseases. An institution that evolved from the Scripps Metabolic Clinic founded by philanthropist Ellen Browning Scripps in 1924, the institute now employs more than 2,500 people on its campuses in La Jolla, CA, and Jupiter, FL, where its renowned scientists including two Nobel laureates and 20 members of the National Academies of Science, Engineering or Medicine work toward their next discoveries. The institutes graduate program, which awards PhD degrees in biology and chemistry, ranks among the top ten of its kind in the nation. In October 2016, TSRI announced a strategic affiliation with the
Most recent headlines
04/08/2024
Dalet, a leading technology and service provider for media-rich organizations, is excited to announce Santiago Solanas as its new Chief Executive Officer (CEO)....
03/06/2024
Dalet, a leading technology and service provider for media-rich organizations, a...
06/05/2024
By Ilyse McKimmie
Now, more than ever
That's a phrase so often used in the last few years that I've come to dread seeing it in notes like this one. A...
06/05/2024
alt= class=wp-image-12099 data-lazy-src=/wp-content/uploads/2024/05/Blog-Exabyte-Storage-Demand-960x540-1.jpg/> Demand for storage solutions has reached unprece...
06/05/2024
Around the world, Asian and Pacific Islander (API) artists continue to impact mu...
06/05/2024
Spotify is making it easier for booklovers to count down the days, hours, minutes, and seconds until a new audiobook releases. With Countdown Pages for audioboo...
06/05/2024
Get Ready to join Dan Hong as he hits the streets in his ultimate culinary journ...
06/05/2024
In this video, cinematographer Simon Rowling welcomes viewers behind the scenes as he lights a daytime-interior scene inside a coffee shop. Shooting on Panavisi...
06/05/2024
L3Harris is well positioned to support the complex and multifaceted nature of special operations forces in all domains through our agile and responsive technolo...
06/05/2024
OAKVILLE, Ontario As part of Emergency Preparedness Week, Alert Ready, Canadas national public alerting system, will be distributing a test alert to Canadians i...
06/05/2024
NEW ROCHELLE, N.Y. Horowitz Research has released a new study on the viewing and media habits of U.S. Hispanic/Latine audiences that shows a dramatic decline in...
06/05/2024
RE:Vision Effects Autograph 2024.4 released! 50% Off Through May 9th
Brie Clayton May 6, 2024
0 Comments
New game-changing motion graphics & VFX featu...
06/05/2024
NBC has renewed Night Court for a third season. The courtroom comedy was on the network from 1984 to 1992, and NBC rebooted it in early 2023....
06/05/2024
ABC has revealed its summer schedule. The Bachelorette gets going Monday, July 8, with Jenn Tran the star in season 21. Celebrity Family Feud starts up Tuesday,...
06/05/2024
It's almost go-time for the Holly Springs Salamanders! The season opener is ...
06/05/2024
06 May 2024
AI and Big Data Take the Centre Stage in Central Asia at Beetech 20...
06/05/2024
FIRST LOOK AT JIM PARSONS & MAYIM BIALIK REPRISING THEIR ROLES FROM THE BIG BAN...
06/05/2024
NBC Sports and Churchill Downs Extend Historic Partnership, Kentucky Derby to Be...
06/05/2024
Beverly Hills Aerials, LIV Golf Make Australian-Broadcast History by Flying Live...
06/05/2024
Back to All News
Watch the Trailer for A Part of You - Felicia Maxime, Edvin Ry...
06/05/2024
Leadership in 12G-SDI for low impact migration to 4k production...
06/05/2024
Press release - 6 May 2024 07:31
STATEMENT CONCERNING EUTELSAT GROUP'S GROUND NETWORK Facebook
LinkedIn
Twitter
Download as PDF
Paris, 06 May 2024 ...
06/05/2024
Tyngsboro, Mass. - May 6, 2024 - Join us at InfoComm 2024 to experience the latest advancements in live production technology. From June 12-14, 2024, at the Las...
06/05/2024
To champion global movements to protect and advance equality for LGBTQ+ communities, Apple is introducing a new Apple Watch Pride Edition Braided Solo Loop, ava...
06/05/2024
Actress Matseliso Mohale has joined Muvhango'Talented actress Matseliso Mohale has joined SABC2's soapie Muvhango' as Vhangani's lover and a c...
06/05/2024
Tonight on Smoke and Mirrors: Leroy refuses to give up fighting to bury MthethoDon't miss Monday, 6 May's riveting episode of South African soapie Smoke...
06/05/2024
Intrinsic, a software and AI robotics company at Alphabet, has integrated NVIDIA AI and Isaac platform technologies to advance the complex field of autonomous r...
06/05/2024
Cybersecurity experts at the RSA Conference this week will be on the hunt for ways to secure their operations in the era of generative AI.
They'll find man...
06/05/2024
1) What is the rationale of the transaction? What is the benefit for SES shareholders?
This combination creates a stronger and more competitive multi-orbit ope...
05/05/2024
A special evening performance by multiplatinum singer Becky G will provide a memorable conclusion to TeievisaUnivision's Casa Cultura upfront event on May 1...
05/05/2024
Giant media buyer Publicis is telling network sales executives that it does not think Nielsen's new panel-plus-big-data currency is ready for this upfront....
05/05/2024
WASHINGTON Glenn Kirschner, legal analyst at NBC News, blasted the media for referring to the New York trial of former President Donald Trump as a hush-money ...
05/05/2024
Catchy Comedy's weekend marathon sees The Beverly Hillbillies on the weekend of May 4-5. The event kicks off Saturday, May 4 at 11 a.m. ET, and runs through...
05/05/2024
NBC Sports has the 150th Kentucky Derby Saturday, May 4. The coverage begins at 2:30 p.m. ET on NBC and Peacock and the feature race at Churchill Downs in Louis...
05/05/2024
Back to All News
Netflix Announces New Comedy Series Roosters, Starring Jeroen ...
05/05/2024
Back to All News
Showdown Alert: Baki Hanma VS Kengan Ashura' Drops Electr...
04/05/2024
NEW YORK As the Asian Pacific American Heritage Month kicks off in May, Nielsen has released an extensive new report diving into their media habits with data sh...
04/05/2024
PHILADELPHIA As National Military Appreciation Month gets underway, Comcast has announced several new initiatives to help veterans, service members, and their f...
04/05/2024
WASHINGTON, D.C. Federal Communications Commission chairwoman Jessica Rosenworcel has announced a tentative agenda for the May Open Commission Meeting scheduled...
04/05/2024
The Broadband Forum has announced major enhancements to key 5G convergence standards that the group said will help advance next-generation applications, improve...
04/05/2024
Spotify Wrapped Campaign Spot Graded With DaVinci Resolve Studio
Brie Clayton May 3, 2024
0 Comments
Blackmagic Design today announced that Colorist M...
04/05/2024
Three Boston Conservatory at Berklee Alums Nominated for Tony Awards An additional eight alums and two current students performed in nominated productions.
...
04/05/2024
New faces and new storylines': What to expect on Izingane ZeS'thembu...
03/05/2024
An innovative learning tool to help media and civil society better understand ho...
03/05/2024
PARK CITY, UTAH - JANUARY 21: (L-R) Actors K stutis Cic nas and Greta Grinevi i t , Director Marija Kavtaradze and Producer Marija Razgute attend the 2023 Sunda...
03/05/2024
If you aren't familiar with Danny Ocean's music, it's only a matter ...
03/05/2024
Spotify has an unwavering commitment to supporting emerging artists across all genres, to helping them launch and thrive in their careers, and to connecting the...
03/05/2024
Como uno de los sonidos de mayor expansi n en el mundo, la M sica Mexicana va m ...
03/05/2024
As one of the fastest-growing sounds worldwide, M sica Mexicana is more than jus...
Firefly Gene Illuminates Ability of Optimized CRISPR-Cpf1 to Efficiently Edit Human Genome JUPITER, FL June 19, 2017 Scientists on the Florida campus of The Scripps Research Institute (TSRI) have improved a state-of-the-art gene-editing technology to advance the system's ability to target, cut and paste genes within human and animal cells and broadening the ways the CRISPR-Cpf1 editing system may be used to study and fight human diseases. 
