Making sense of heritable human genome editing: Scientific and ethical considerations

Greenfield, Andy

doi:10.1016/bs.pmbts.2020.12.008

Cited by 8 publications

(4 citation statements)

References 38 publications

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“…While the CRISPR/Cas9 system has demonstrated a great promise for a variety of applications, there are several factors that influence its efficacy as well as its safety which must be addressed, especially when the goal is in vivo human gene therapy. Last but not least, there are ethical implications that need to be carefully considered, and are discussed extensively elsewhere (118)(119)(120). A more worrisome problem includes specific biosafety regulatory challenges and ethical issues concerning applications of CRISPR technology for irreversibly editing the human genome.…”

Section: Advantages and Challenges Of Crispr-mediated Transcriptional...mentioning

confidence: 99%

“…A more worrisome problem includes specific biosafety regulatory challenges and ethical issues concerning applications of CRISPR technology for irreversibly editing the human genome. Nevertheless, ethical concerns need to be addressed properly, which may not be unique when considering other interventions that influence human biology (119).…”

Section: Advantages and Challenges Of Crispr-mediated Transcriptional...mentioning

confidence: 99%

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Tailoring Cardiac Synthetic Transcriptional Modulation Towards Precision Medicine

Schoger

Panàkovà

Zelarayán

2022

Front. Cardiovasc. Med.

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Molecular and genetic differences between individual cells within tissues underlie cellular heterogeneities defining organ physiology and function in homeostasis as well as in disease states. Transcriptional control of endogenous gene expression has been intensively studied for decades. Thanks to a fast-developing field of single cell genomics, we are facing an unprecedented leap in information available pertaining organ biology offering a comprehensive overview. The single-cell technologies that arose aided in resolving the precise cellular composition of many organ systems in the past years. Importantly, when applied to diseased tissues, the novel approaches have been immensely improving our understanding of the underlying pathophysiology of common human diseases. With this information, precise prediction of regulatory elements controlling gene expression upon perturbations in a given cell type or a specific context will be realistic. Simultaneously, the technological advances in CRISPR-mediated regulation of gene transcription as well as their application in the context of epigenome modulation, have opened up novel avenues for targeted therapy and personalized medicine. Here, we discuss the fast-paced advancements during the recent years and the applications thereof in the context of cardiac biology and common cardiac disease. The combination of single cell technologies and the deep knowledge of fundamental biology of the diseased heart together with the CRISPR-mediated modulation of gene regulatory networks will be instrumental in tailoring the right strategies for personalized and precision medicine in the near future. In this review, we provide a brief overview of how single cell transcriptomics has advanced our knowledge and paved the way for emerging CRISPR/Cas9-technologies in clinical applications in cardiac biomedicine.

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Section: Advantages and Challenges Of Crispr-mediated Transcriptional...mentioning

confidence: 99%

Section: Advantages and Challenges Of Crispr-mediated Transcriptional...mentioning

confidence: 99%

Tailoring Cardiac Synthetic Transcriptional Modulation Towards Precision Medicine

Schoger

Panàkovà

Zelarayán

2022

Front. Cardiovasc. Med.

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“…Somatic genome editing is well suited for therapeutic purposes of monogenic inherited disorders in which mutations in a single gene are the cause of diseases often causing premature death and for which effective treatment options are not available; for example, cystic fibrosis and severe combined immunodeficiency [3]. Therapeutic applications of SGE have had notable successes with T-cell therapies, which offer great promise in cancer treatment [4]; more recently, SGE has been used to cure patients suffering from a form of retinal blindness called Leber congenital amaurosis, and as treatment for the blood disorder haemophilia B…”

Section: Introductionmentioning

confidence: 99%

Somatic Genome Editing: Technical Challenges and Ethical Appraisal

O’Keeffe,

Alphonse,

Mendz

2024

ejmhr

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The ability to modify the DNA sequences with molecular tools that employ nucleases has made it possible to edit genomes. Somatic genome editing is the procedure to alter the genome of somatic cells, making the changes introduced into the nucleotide sequence not inheritable. Powerful tools have been developed for therapeutic purposes to correct monogenic inherited disorders that often cause premature death and for which effective treatment options are not available. To ensure positive impact and minimise potential harms of these techniques, require to understand their limits in addition to apply values and principles that guide their use. This study reviews technical challenges of genome editing and appraises the Ethics of its application.

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“…subsidiary legislation) relating to the use of assisted reproductive technology (ART). [3] Given that HHGE can be viewed as a new ART, [4] it can be regulated as such. Regulating HHGE as an ART offers a fast track to the legislative agenda for HHGE.…”

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confidence: 99%

A golden opportunity for South Africa to legislate on human heritable genome editing

Thaldar

2023

S Afr J Bioethics Law

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Background. South Africa (SA) currently has a golden opportunity to legislate on human heritable genome editing (HHGE), as the country is revising its assisted reproductive technology regulations. A set of sub-regulations that deals with HHGE, which could seamlessly slot into the current regulations, has already been developed. The principles underlying the proposed set of sub-regulations are as follows: HHGE should be regulated to improve the lives of the people and should not be banned; the well-established standard of safety and efficacy should be used in the regulation of HHGE; quality of life is what matters, and not preserving a ‘normal’ genome; parents’ reproductive autonomy must be respected; and equality of access to approved HHGE technology should be promoted. Objectives. To ascertain whether the proposed set of sub-regulations that deals with HHGE is aligned with public opinion in SA, and SA’s Bill of Rights. Methods. Public opinion in SA is determined with reference to a deliberative public engagement study on HHGE conducted among South Africans, and the relevant rights in the Bill of Rights are interpreted with reference to recent case law. Results. This proposed set of sub-regulations that deals with HHGE is aligned with public opinion in SA, and SA’s Bill of Rights. Conclusion. Despite the legal and ethical complexities of HHGE, the proposed set of sub-regulations provides a targeted and effective legislative approach. They fit seamlessly into the country’s existing health law framework, creating specific legal standards for HHGE that align with both public opinion and the country’s Bill of Rights.

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Making sense of heritable human genome editing: Scientific and ethical considerations

Cited by 8 publications

References 38 publications

Tailoring Cardiac Synthetic Transcriptional Modulation Towards Precision Medicine

Tailoring Cardiac Synthetic Transcriptional Modulation Towards Precision Medicine

Somatic Genome Editing: Technical Challenges and Ethical Appraisal

A golden opportunity for South Africa to legislate on human heritable genome editing

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