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Rev. bioét. derecho ; (47): 17-31, nov. 2019.
Artigo em Espanhol | IBECS | ID: ibc-184863


La tecnología de la edición genética por CRISPR ha revolucionado tanto la investigación en biotecnología como en biomedicina. Esta técnica tan poderosa y versátil permite editar los genes de cualquier especie a la carta. A pesar de su potencia y efectividad, quedan muchas cuestiones para resolver y controlar su resultado final, particularmente en las aplicaciones sobre el genoma de los seres humanos. En este artículo se plantean tanto los puntos fuertes y puntos débiles de la técnica, como otras cuestiones abiertas sobre la edición génica, sobre si debe dirigirse a la terapia o a la mejora, si la modificación debe constreñirse a células somáticas o también a editar a embriones, modificando el genoma de los seres humanos del futuro

La tecnologia de l'edició gènica mitjançant CRISPR ha revolucionat tant la recerca en biotecnologia com en biomedicina. Aquesta tècnica tan poderosa i versàtil permet editar els gens de qualsevol espècie a la carta. Malgrat la seva potència i efectivitat, queden moltes qüestions per resoldre i controlar el seu resultat final, particularment en les aplicacions sobre el genoma dels éssers humans. En aquest article es plantegen els punts forts i els punts febles de la tècnica, així com altres qüestions obertes sobre l'edició gènica, si ha de dirigir-se a la teràpia o a la millora, si la modificació ha de constrènyer-se a cèl•lules somàtiques o també es poden editar en embrions, modificant el genoma dels éssers humans del futur

Gene using CRISPR has completely revolutionized the research in biotechnology and biomedicine. This powerful and versatile technique enables the precise edition of genes from any organism. Even though the technique is so effective and amenable, many questions remain to be solved before the final genetic outcome can be fully controlled, particularly in its uses on the human genome. In this article, I discuss the current strengths and weaknesses of the technique. I also pose other open questions on gene editing, such as whether it should be used for either therapy or genetic enhancement, and whether it should be used only on somatic cells or also for embryo gene editing, the latter resulting in the modification of future human beings

Humanos , Edição de Genes/tendências , Sistemas CRISPR-Cas , Terapia Genética/legislação & jurisprudência , Tecnologia de Impulso Genético/legislação & jurisprudência , Repetições Palindrômicas Curtas Agrupadas e Regularmente Espaçadas , Células Germinativas
Life Sci ; 232: 116636, 2019 Sep 01.
Artigo em Inglês | MEDLINE | ID: mdl-31295471


Till date, only three techniques namely Zinc Finger Nuclease (ZFN), Transcription-Activator Like Effector Nucleases (TALEN) and Clustered Regularly Interspaced Short Palindromic Repeats-CRISPR-Associated 9 (CRISPR-Cas9) are available for targeted genome editing. CRISPR-Cas system is very efficient, fast, easy and cheap technique for achieving knock-out gene in the cell. CRISPR-Cas9 system refurbishes the targeted genome editing approach into a more expedient and competent way, thus facilitating proficient genome editing through embattled double-strand breaks in approximately any organism and cell type. The off-target effects of CRISPR Cas system has been circumnavigated by using paired nickases. Moreover, CRISPR-Cas9 has been used effectively for numerous purposes, like knock-out of a gene, regulation of endogenous gene expression, live-cell labelling of chromosomal loci, edition of single-stranded RNA and high-throughput gene screening. The execution of the CRISPR-Cas9 system has amplified the number of accessible scientific substitutes for studying gene function, thus enabling generation of CRISPR-based disease models. Even though many mechanistic questions are left behind to be answered and the system is not yet fool-proof i.e., a number of challenges are yet to be addressed, the employment of CRISPR-Cas9-based genome engineering technologies will increase our understanding to disease processes and their treatment in the near future. In this review we have discussed the history of CRISPR-Cas9, its mechanism for genome editing and its application in animal, plant and protozoan parasites. Additionally, the pros and cons of CRISPR-Cas9 and its potential in therapeutic application have also been detailed here.

Sistemas CRISPR-Cas , Edição de Genes/métodos , Animais , Repetições Palindrômicas Curtas Agrupadas e Regularmente Espaçadas , Edição de Genes/tendências , Genoma , Humanos , Plantas/genética
Transgenic Res ; 28(Suppl 2): 57-60, 2019 08.
Artigo em Inglês | MEDLINE | ID: mdl-31321684


Animal husbandry is believed to predate farming of crops, and remains a core component of most agricultural systems. Historic breeding strategies were based largely on visual observation, crossing animals that were perceived to display enhanced merit. Advances in sequencing capacity coupled with reduced costs have allowed genomic selection tools to deliver significant contribution to breeding regimes. The application of genome editors to make specific changes to livestock genomes has the potential to deliver additional benefits.

Produtos Agrícolas/genética , Edição de Genes/tendências , Genômica , Criação de Animais Domésticos/tendências , Cruzamento , Produtos Agrícolas/crescimento & desenvolvimento , Engenharia Genética/tendências , Genoma/genética , Humanos
Transgenic Res ; 28(Suppl 2): 61-64, 2019 08.
Artigo em Inglês | MEDLINE | ID: mdl-31321685


Processes of traditional trait development in plants depend on genetic variations derived from spontaneous mutation or artificial random mutagenesis. Limited availability of desired traits in crossable relatives or failure to generate the wanted phenotypes by random mutagenesis led to develop innovative breeding methods that are truly cross-species and precise. To this end, we devised novel methods of precise genome engineering that are characterized to use pre-assembled CRISPR/Cas9 ribonucleoprotein (RNP) complex instead of using nucleic ands or Agrobacterium. We found that our methods successfully engineered plant genomes without leaving any foreign DNA footprint in the genomes. To facilitate introduction of RNP into plant nucleus, we first obtained protoplasts after removing the transfection barrier, cell wall. Whole plants were regenerated from the single cell of protoplasts that has been engineered with the RNP. Pending the improved way of protoplast regeneration technology especially in crop plants, our methods should help develop novel traits in crop plants in relatively short time with safe and precise way.

Sistemas CRISPR-Cas/genética , DNA/genética , Edição de Genes/tendências , Ribonucleoproteínas/genética , Agrobacterium/genética , Genoma de Planta/genética , Mutação , Protoplastos/metabolismo
Transgenic Res ; 28(Suppl 2): 151-159, 2019 08.
Artigo em Inglês | MEDLINE | ID: mdl-31321698


Australia's gene technology regulatory scheme (GT Scheme) regulates activities with genetically modified organisms (GMOs, organisms modified by gene technology), including environmental releases. The scope of regulation, i.e. what organisms are and are not regulated, is set by the Gene Technology Act 2000 (GT Act) and GT Regulations 2001 (GT Regulations). The GT Act gives broad, overarching definitions of 'gene technology' and 'GMO' but also provides for exclusions and inclusions in the GT Regulations. Whether organisms developed with genome editing techniques are, or should be, regulated under countries' national GMO laws is the subject of debate globally. These issues are also under active consideration in Australia. A technical review of the GT Regulations was initiated in 2016 to clarify the regulatory status of genome editing. Proposed draft amendments are structured around whether the process involves introduction of a nucleic acid template. If agreed, amendments would exclude from regulation organisms produced using site directed nuclease (SDN) 1 techniques while organisms produced using oligonucleotide mutagenesis, SDN-2 or SDN-3 would continue to be regulated as GMOs. The review of the GT Regulations is still ongoing and no legislative changes have been made to the GT Regulations. A broader policy review of the GT Scheme was undertaken in 2017-2018 and as a result further work will be undertaken on the scope and definitions of the GT Act in light of ongoing developments.

Alimentos Geneticamente Modificados , Edição de Genes/tendências , Engenharia Genética/tendências , Organismos Geneticamente Modificados/genética , União Europeia , Humanos
Transgenic Res ; 28(Suppl 2): 165-168, 2019 08.
Artigo em Inglês | MEDLINE | ID: mdl-31321700


The development of gene editing techniques, capable of producing plants and animals with new and improved traits, is revolutionizing the world of plant and animal breeding and rapidly advancing to commercial reality. However, from a regulatory standpoint the Government of Canada views gene editing as another tool that will join current methods used to develop desirable traits in plants and animals. This is because Canada focusses on the potential risk resulting from the novelty of the trait, or plant or animal product entering the Canadian environment or market place, rather than the process or method by which it was created. The Canadian Food Inspection Agency is responsible for the regulation of the environmental release of plants with novel traits, and novel livestock feeds, while Health Canada is responsible for the regulation of novel foods. Environment and Climate Change Canada, in partnership with Health Canada, regulates modified animals for entry into the environment. In all cases, these novel products may be the result of conventional breeding, mutagenesis, recombinant DNA techniques or other methods of plant or animal breeding such as gene editing. This novelty approach allows the Canadian regulatory system to efficiently adjust to any new developments in the science of plant and animal breeding and allows for risk-appropriate regulatory decisions. This approach encourages innovation while maintaining science-based regulatory expertise. Canadian regulators work cooperatively with proponents to determine if their gene editing-derived product meets the definition of a novel product, and whether it would be subject to a pre-market assessment. Therefore, Canada's existing regulatory system is well positioned to accommodate any new innovations or technologies in plant or animal breeding, including gene editing.

Produtos Agrícolas/genética , Edição de Genes/tendências , Engenharia Genética/legislação & jurisprudência , Genoma de Planta/genética , Animais , Canadá , Produtos Agrícolas/crescimento & desenvolvimento , Alimentos Geneticamente Modificados , Edição de Genes/legislação & jurisprudência , Gado/genética , Gado/crescimento & desenvolvimento , Melhoramento Vegetal/legislação & jurisprudência , Plantas Geneticamente Modificadas/genética , Plantas Geneticamente Modificadas/crescimento & desenvolvimento
Transgenic Res ; 28(Suppl 2): 175-181, 2019 08.
Artigo em Inglês | MEDLINE | ID: mdl-31321702


In India, genetically modified organisms and products thereof are regulated under the "Rules for the manufacture, use, import, export and storage of hazardous microorganisms, genetically engineered organisms or cells, 1989" (referred to as Rules, 1989) notified under the Environment (Protection) Act, 1986. These Rules are implemented by the Ministry of Environment, Forest and Climate Change, Department of Biotechnology and State Governments though six competent authorities. The Rules, 1989 are supported by series of guidelines on contained research, biologics, confined field trials, food safety assessment, environmental risk assessment etc. The definition of genetic engineering in the Rules, 1989 implies that new genome engineering technologies including gene editing technologies like CRISPR/Cas9 and gene drives may be covered under the rules. The regulatory authorities if required, may also review the experiences of other countries in dealing with such new and emerging technologies.

Biotecnologia/tendências , Sistemas CRISPR-Cas/genética , Edição de Genes/tendências , Plantas Geneticamente Modificadas/genética , Inocuidade dos Alimentos , Humanos , Índia , Plantas Geneticamente Modificadas/crescimento & desenvolvimento
Yi Chuan ; 41(7): 582-598, 2019 Jul 20.
Artigo em Chinês | MEDLINE | ID: mdl-31307968


Gene editing is a genetic manipulation technology which utilizes bacterial nucleases to accurately and efficiently modify DNA or RNA. Gene editing has broad applications in basic research, breeding, and drug screening, and it is gaining validity and applicability to the therapy of many diseases especially genetic-based disease. In this review, we summarize the development of gene editing technology, its different strategies and applications in the treatment of disease, and the research of gene editing therapy for genetic diseases (including base editor and epigenetic regulation) in the treatment of disorders and diseases of the blood system, liver, muscle and nervous system. Finally, we discuss the future development prospects of gene editing therapy.

Sistemas CRISPR-Cas , Edição de Genes/tendências , Terapia Genética , Doença , Epigênese Genética , Humanos
BMB Rep ; 52(8): 475-481, 2019 Aug.
Artigo em Inglês | MEDLINE | ID: mdl-31234957


The evolution of genome editing technology based on CRISPR (clustered regularly interspaced short palindromic repeats) system has led to a paradigm shift in biological research. CRISPR/Cas9-guide RNA complexes enable rapid and efficient genome editing in mammalian cells. This system induces double-stranded DNA breaks (DSBs) at target sites and most DNA breakages induce mutations as small insertions or deletions (indels) by non-homologous end joining (NHEJ) repair pathway. However, for more precise correction as knock-in or replacement of DNA base pairs, using the homology-directed repair (HDR) pathway is essential. Until now, many trials have greatly enhanced knock-in or substitution efficiency by increasing HDR efficiency, or newly developed methods such as Base Editors (BEs). However, accuracy remains unsatisfactory. In this review, we summarize studies to overcome the limitations of HDR using the CRISPR system and discuss future direction. [BMB Reports 2019; 52(8): 475-481].

Proteína 9 Associada à CRISPR/genética , Repetições Palindrômicas Curtas Agrupadas e Regularmente Espaçadas/genética , Edição de Genes/tendências , Genoma/genética , Animais , Proteína 9 Associada à CRISPR/metabolismo , Humanos
Eur J Med Genet ; 62(8): 103682, 2019 Aug.
Artigo em Inglês | MEDLINE | ID: mdl-31150829


Perhaps the two most significant pioneering biomedical discoveries with immediate clinical implications during the past forty years have been the advent of assisted reproductive technologies (ART) and the genetics revolution. ART, including in vitro fertilization (IVF), intracytoplasmic sperm injection and preimplantation genetic testing, has resulted in the birth of more than 8 million children, and the pioneer of IVF, Professor Bob Edwards, was awarded the 2010 Nobel Prize. The genetics revolution has resulted in our genomes being sequenced and many of the molecular mechanisms understood, and technologies for genomic editing have been developed. With the combination of nearly routine ART protocols for healthy conceptions together with almost error-free, inexpensive and simple methods for genetic modification, the question "Are we ready for genome editing in human embryos for clinical purposes?" was debated at the 5th congress on controversies in preconception, preimplantation and Prenatal Genetic Diagnosis, in collaboration with the Ovarian Club Meeting, in November 2018 in Paris. The co-authors each presented scientific, medical and bioethical backgrounds, and the debate was chaired by Professor Alan Handyside. In this paper, we consider whether genome editing is safe and ethical. We conclude that we are currently not ready for genome editing to be used in human embryos for clinical purposes, and we call for a global debate to determine if and when this technology could be used in ART. ‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬.

Fertilização In Vitro/tendências , Edição de Genes/tendências , Diagnóstico Pré-Implantação/tendências , Técnicas de Reprodução Assistida , Feminino , Testes Genéticos , Humanos , Gravidez , Injeções de Esperma Intracitoplásmicas
Med Sci (Paris) ; 35(4): 309-315, 2019 Apr.
Artigo em Francês | MEDLINE | ID: mdl-31038108


The idea according to which the most recent therapeutic methods will overcome the more traditional pharmacopoeia is widespread in recent publications. Biomedicine and gene therapies are booming, but we realize, as for other therapeutic approaches, that they suffer intrinsic constraints and limitations and that their most relevant therapeutic fields are complementary to those of traditional drugs. They are now viewed as potentially synergistic with these traditional drugs, rather than competitors. This review puts into perspective the potential of genome editing in the field of drug discovery and therapeutic innovation.

Descoberta de Drogas , Edição de Genes , Terapias em Estudo/tendências , Animais , Sistemas CRISPR-Cas/genética , Descoberta de Drogas/história , Descoberta de Drogas/métodos , Descoberta de Drogas/tendências , Edição de Genes/história , Edição de Genes/tendências , Terapia Genética/história , Terapia Genética/métodos , Terapia Genética/tendências , História do Século XX , História do Século XXI , Humanos , Terapias em Estudo/métodos