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The Council of Europe should not reaffirm the ban on germline genome editing in humans.
The Council of Europe plans to urge member states to sign and ratify the Oviedo Convention that would ban all inheritable modifications of the human germline. Such a policy would prevent research to develop new therapeutic options for inheritable diseases in Europe and is in sharp contrast to international developments. [Image: see text]
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Targeted gene disruption by CRISPR/xCas9 system in Drosophila melanogaster.
Although the Cas9 protein from Streptococcus pyogenes (SpCas9) is the most widely used clustered regularly interspaced short palindromic repeats (CRISPR) variant in genome engineering experiments, it does have certain limitations. First, the stringent requirement for the protospacer adjacent motif (PAM) sequence limits the target DNA that can be manipulated using this method in insects. Second, its complementarity specifications are not very stringent, meaning that it can sometimes cause off-target effects at the target site. A recent study reported that an evolved SpCas9 variant, xCas9(3.7), with preference for various 5'-NG-3' PAM sequences not only has the broadest PAM compatibility but also has much greater DNA specificity and lower genome-wide off-target activity than SpCas9 in mammalian cells. Here we applied the CRISPR/xCas9 system to target the white gene in Drosophila melanogaster, testing the genome-editing efficiency of xCas9 at different PAM sites. On the GGG PAM site, xCas9 showed less activity than SpCas9. For the non-NGG PAM site TGA, xCas9 could produce DNA cleavage and indel-mediated disruption on the target gene. However, for other non-NGG PAM sites, xCas9 showed no activity. These findings show that the evolved Cas9 variant with broad PAM compatibility is functional in Drosophila to induce heritable gene alterations, increasing the targeting range for the applications of genome editing in insects.
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Genetically Engineering the Nervous System with CRISPR-Cas.
The multitude of neuronal subtypes and extensive interconnectivity of the mammalian brain presents a substantial challenge to those seeking to decipher its functions. While the molecular mechanisms of several neuronal functions remain poorly characterized, advances in next-generation sequencing (NGS) and gene-editing technology have begun to close this gap. The clustered regularly interspaced short palindromic repeats (CRISPR)-associated protein (CRISPR-Cas) system has emerged as a powerful genetic tool capable of manipulating the genome of essentially any organism and cell type. This technology has advanced our understanding of complex neurologic diseases by enabling the rapid generation of novel, disease-relevant in vitro and transgenic animal models. In this review, we discuss recent developments in the rapidly accelerating field of CRISPR-mediated genome engineering. We begin with an overview of the canonical function of the CRISPR platform, followed by a functional review of its many adaptations, with an emphasis on its applications for genetic interrogation of the normal and diseased nervous system. Additionally, we discuss limitations of the CRISPR editing system and suggest how future modifications to existing platforms may advance our understanding of the brain.
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CRISPR, Patents, and the Public Health.
Patent issues surrounding CRISPR, the revolutionary genetic editing technology, may have important implications for the public health. Patents maintain high prices for novel therapies, limiting patient access. Relatedly, insurance coverage for expensive therapies is waning. Patents also misallocate research and development resources to profitable disease indications rather than those that necessarily impinge on the public health. And it is unclear how CRISPR therapies will figure into the current regulatory framework for biosimilars. Policy makers and physicians should consider these issues now, before CRISPR therapies become widely adopted-and entrenched-in the marketplace.
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Cancer: Can CRISPR Become a Game Changer?
Recent development in CRISPR technologies has emerged as a powerful tool for genome editing, which can become a powerful diagnostic and therapeutic option for cancer patients. While literature search demonstrates some successes in this dimension in animal models and phase-I human trials, still the promised panacea has shortcomings in terms of off-target mutations (OTMs), minimal efficiency, and tailoring individualised interventions. While there is a promise for the future, although the CRISPR technology still has to climb up the ladder in terms of improvements with more enhanced efficiency tools for delivering the payload into cell and reducing the off-target mutations. Key Words: CRISPR, Oncology, Off-target mutations (OTMs), Genome editing, 3-D cell cultures.
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CRISPR Cautions: Biosecurity Implications of Gene Editing.
CRISPR, a powerful gene-editing technology, is revolutionizing the life sciences and medical research. The technology has also become democratized. Costs to use CRISPR are low and decreasing, kits are available to make the use of CRISPR straightforward, and there is a rapidly growing scientific literature describing CRISPR methodologies and novel applications. However, like other powerful advances in the life sciences, CRISPR raises biosecurity concerns: it could be misused for harm, and it lowers technical barriers to biological weapons development. This essay describes the history and dissemination of CRISPR as genome-editing techniques have become widespread, outlines potential biosecurity concerns, and recommends actions governments and scientists may take to reduce biosecurity risks. While it is not possible to eliminate biosecurity risks from the misuse of biotechnologies, including CRISPR, steps can be taken to increase security while allowing this powerful technology to remain widely available for beneficent purposes.
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NEW BIOTECHNOLOGICAL TREATMENTS FOR LIPID DISORDERS.
Disorders of lipid and lipoprotein metabolism play a central role in the pathogenesis of atherosclerotic cardiovascular diseases (CVDs). Despite the widespread use of efficacious lipid-modifying therapies, the residual risk of CVD remains unacceptably high. The purpose of this manuscript is to review the application of new technologies in the treatment of lipid disorders. New therapies work mostly at the gene expression level and are, therefore, different from traditional small-molecule drugs that work mainly by inhibiting already synthesized proteins. We will briefly lay out the function of the gene products targeted by the new agents. Then, we will organize our review of new biotechnological treatments by the molecular approach, namely: monoclonal antibodies, antisense oligonucleotides, small-interfering RNAs, and Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated 9 (Cas9)-based genome editing. The paper concludes with the description of the current clinical studies and the perspectives for the use of these agents. (REV INVEST CLIN. 2018;70:244-54).
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In dogs, CRISPR fixes a muscular dystrophy.
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CRISPR-Cas in Medicinal Chemistry: Applications and Regulatory Concerns.
A rapid search in scientific publication's databases shows how the use of CRISPR-Cas genome editions' technique has considerably expanded, and its growing importance, in modern molecular biology. Just in pub-med platform, the search of the term gives more than 3000 results. Specifically, in Drug Discovery, Medicinal Chemistry and Chemical Biology in general CRISPR method may have multiple applications. Some of these applications are: resistance-selection studies of antimalarial lead organic compounds; investigation of druggability; development of animal models for chemical compounds testing, etc. In this paper, we offer a review of the most relevant scientific literature illustrated with specific examples of application of CRISPR technique to medicinal chemistry and chemical biology. We also present a general overview of the main legal and ethical trends regarding this method of genome editing.
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CRISPR/Cas9-mediated genome editing in plants.
The increasing burden of the world's population on agriculture necessitates the development of more robust crops. As the amount of information from sequenced crop genomes increases, technology can be used to investigate the function of genes in detail and to design improved crops at the molecular level. Recently, an RNA-programmed genome-editing system composed of a clustered regularly interspaced short palindromic repeats (CRISPR)-encoded guide RNA and the nuclease Cas9 has provided a powerful platform to achieve these goals. By combining versatile tools to study and modify plants at different molecular levels, the CRISPR/Cas9 system is paving the way towards a new horizon for basic research and crop development. In this review, the accomplishments, problems and improvements of this technology in plants, including target sequence cleavage, knock-in/gene replacement, transcriptional regulation, epigenetic modification, off-target effects, delivery system and potential applications, will be highlighted.
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