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The code breaker : Jennifer Doudna, gene editing, and the future of the human race
\"The bestselling author of Leonardo da Vinci and Steve Jobs returns with a gripping account of how the pioneering scientist Jennifer Doudna, along with her colleagues and rivals, launched a revolution that will allow us to cure diseases, fend off viruses, and enhance our children\"-- Provided by publisher.
The promise and challenge of therapeutic genome editing
Genome editing, which involves the precise manipulation of cellular DNA sequences to alter cell fates and organism traits, has the potential to both improve our understanding of human genetics and cure genetic disease. Here I discuss the scientific, technical and ethical aspects of using CRISPR (clustered regularly interspaced short palindromic repeats) technology for therapeutic applications in humans, focusing on specific examples that highlight both opportunities and challenges. Genome editing is—or will soon be—in the clinic for several diseases, with more applications under development. The rapid pace of the field demands active efforts to ensure that this breakthrough technology is used responsibly to treat, cure and prevent genetic disease.
The scientific, technical and ethical aspects of using CRISPR technology for therapeutic applications in humans are discussed, highlighting both opportunities and challenges of this technology to treat, cure and prevent genetic disease.
Journal Article
The mutant project : inside the global race to genetically modify humans
As scientists elsewhere start to catch up with China's vast genetic research programme, gene editing is fuelling an innovation economy that threatens to widen racial and economic inequality. Fundamental questions about science, health and social justice are at stake. Who gets access to gene-editing technologies? As countries loosen regulations around the globe, can we shape research agendas to promote an ethical and fair society? Professor Eben Kirksey takes us on a groundbreaking journey to meet the key scientists, lobbyists and entrepreneurs who are bringing cutting-edge genetic modification tools like CRISPR to your local clinic.
Book
Various Aspects of a Gene Editing System—CRISPR–Cas9
The discovery of clustered, regularly interspaced short palindromic repeats (CRISPR) and their cooperation with CRISPR-associated (Cas) genes is one of the greatest advances of the century and has marked their application as a powerful genome engineering tool. The CRISPR–Cas system was discovered as a part of the adaptive immune system in bacteria and archaea to defend from plasmids and phages. CRISPR has been found to be an advanced alternative to zinc-finger nucleases (ZFN) and transcription activator-like effector nucleases (TALEN) for gene editing and regulation, as the CRISPR–Cas9 protein remains the same for various gene targets and just a short guide RNA sequence needs to be altered to redirect the site-specific cleavage. Due to its high efficiency and precision, the Cas9 protein derived from the type II CRISPR system has been found to have applications in many fields of science. Although CRISPR–Cas9 allows easy genome editing and has a number of benefits, we should not ignore the important ethical and biosafety issues. Moreover, any tool that has great potential and offers significant capabilities carries a level of risk of being used for non-legal purposes. In this review, we present a brief history and mechanism of the CRISPR–Cas9 system. We also describe on the applications of this technology in gene regulation and genome editing; the treatment of cancer and other diseases; and limitations and concerns of the use of CRISPR–Cas9.
Journal Article
Pandemic
\"After a young, seemingly healthy woman collapses suddenly on the NYC subway and dies by the time she reaches the hospital, her case is initially chalked up to a virulent strain of influenza. That is, until she ends up on Dr. Jack Stapleton's autopsy table, where Jack discovers something eerily fishy: first, that the young woman has had a heart transplant, and second, that her DNA matches that of the transplanted heart. Strangely, two more incidences of young people with this same sudden and rapid illness follow, and Jack fears that this could be the start of an unprecedented pandemic. But the facts aren't adding up. Something is off about these cases, something creepy, and only Jack can figure it out before it's too late. Thus begins a race against time, during which Jack unveils the dark underbelly of the organ-transplant market. His name is Bui Zhao, a businessman and hospital board member who has been cheating the system by using organs and cells from chimeric pigs, via the gene-editing biotechnology CRISPER/CAS9, which allows pig genes to be inserted into living human cells. In a climactic mortal showdown, Jack must face Zhao, the megalomaniac willing to risk the fate of the world to purse his commercial interests, if he wants to save the future of medicine\"-- Provided by publisher.
BOOK
CRISPR plants now subject to tough GM laws in European Union
Top court’s ruling threatens research on gene-edited crops in the bloc.
Top court’s ruling threatens research on gene-edited crops in European Union.
Journal Article
Prime editing efficiently generates W542L and S621I double mutations in two ALS genes in maize
Prime editing is a novel and universal CRISPR/Cas-derived precision genome-editing technology that has been recently developed. However, low efficiency of prime editing has been shown in transgenic rice lines. We hypothesize that enhancing pegRNA expression could improve prime-editing efficiency. In this report, we describe two strategies for enhancing pegRNA expression. We construct a prime editing vector harboring two pegRNA variants for W542L and S621I double mutations in
ZmALS1
and
ZmALS2
. Compared with previous reports in rice, we achieve much higher prime-editing efficiency in maize. Our results are inspiring and provide a direction for the optimization of plant prime editors.
Journal Article
CRISPR-Cas9 In Vivo Gene Editing of KLKB1 for Hereditary Angioedema
Hereditary angioedema is a rare genetic disease that leads to severe and unpredictable swelling attacks. NTLA-2002 is an in vivo gene-editing therapy based on clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated protein 9. NTLA-2002 targets the gene encoding kallikrein B1 (
), with the goal of lifelong control of angioedema attacks after a single dose.
In this phase 1 dose-escalation portion of a combined phase 1-2 trial of NTLA-2002 in adults with hereditary angioedema, we administered NTLA-2002 at a single dose of 25 mg, 50 mg, or 75 mg. The primary end points were the safety and side-effect profile of NTLA-2002 therapy. Secondary and exploratory end points included pharmacokinetics, pharmacodynamics, and clinical efficacy determined on the basis of investigator-confirmed angioedema attacks.
Three patients received 25 mg of NTLA-2002, four received 50 mg, and three received 75 mg. At all dose levels, the most common adverse events were infusion-related reactions and fatigue. No dose-limiting toxic effects, serious adverse events, grade 3 or higher adverse events, or clinically important laboratory findings were observed after the administration of NTLA-2002. Dose-dependent reductions in the total plasma kallikrein protein level were observed between baseline and the latest assessment, with a mean percentage change of -67% in the 25-mg group, -84% in the 50-mg group, and -95% in the 75-mg group. The mean percentage change in the number of angioedema attacks per month between baseline and weeks 1 through 16 (primary observation period) was -91% in the 25-mg group, -97% in the 50-mg group, and -80% in the 75-mg group. Among all the patients, the mean percentage change in the number of angioedema attacks per month from baseline through the latest assessment was -95%.
In this small study, a single dose of NTLA-2002 led to robust, dose-dependent, and durable reductions in total plasma kallikrein levels, and no severe adverse events were observed. In exploratory analyses, reductions in the number of angioedema attacks per month were observed at all dose levels. (Funded by Intellia Therapeutics; ClinicalTrials.gov number, NCT05120830.).
Journal Article
UK first to approve CRISPR treatment for diseases: what you need to know
The landmark decision could transform the treatment of sickle-cell disease and β-thalassaemia — but the technology is expensive.
The landmark decision could transform the treatment of sickle-cell disease and β-thalassaemia — but the technology is expensive.
Credit: Eye Of Science/Science Photo Library
Coloured scanning electron micrograph of red blood cells affected by sickle cell anaemia.
Journal Article
Efficient and transgene-free genome editing in wheat through transient expression of CRISPR/Cas9 DNA or RNA
Editing plant genomes is technically challenging in hard-to-transform plants and usually involves transgenic intermediates, which causes regulatory concerns. Here we report two simple and efficient genome-editing methods in which plants are regenerated from callus cells transiently expressing CRISPR/Cas9 introduced as DNA or RNA. This transient expression-based genome-editing system is highly efficient and specific for producing transgene-free and homozygous wheat mutants in the T0 generation. We demonstrate our protocol to edit genes in hexaploid bread wheat and tetraploid durum wheat, and show that we are able to generate mutants with no detectable transgenes. Our methods may be applicable to other plant species, thus offering the potential to accelerate basic and applied plant genome-engineering research.
Plant genome editing typically relies upon transgenic intermediates, which is a concern given the current regulatory requirements concerning GMOs. Here, Zhang
et al
. describe a method to edit wheat genomes by transiently expressing CRISPR/Cas9 DNA or RNA, and are able to generate mutant plants with no detectable transgenes.
Journal Article