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Ethical and Regulatory Considerations for Developing Gene Therapies Involving Genome Editing.
Developing gene therapies involving gene editing is a rapidly evolving field with large potential implications for improving health for both rare and common diseases. Ensuring that these technologies are developed safely, efficiently, and fairly is essential. To better understand the ethical considerations and regulatory requirements and challenges with gene therapies involving gene editing that may advance precision health. Through a multistakeholder workshop and subsequent engagements, multiple ethical and regulatory barriers to developing and implementing gene therapies involving gene editing were identified. Eight major themes emerged that warrant careful consideration, including (1) objectives (treatment, risk reduction, and enhancement) for the intervention; (2) competing interests of safety, equity, and desire for research efficiencies; (3) unique aspects of gene editing related to rare and ultrarare genetic conditions; (4) considerations in the pediatric population; (5) regulatory requirements and ethics oversight; (6) challenges with long-term follow-up and data sharing; and (7) communication. To promote the safe, efficient development of gene therapies involving gene editing that will reach their full potential, all stakeholders will have to undertake an unprecedented degree of collaboration. However, this will be essential to ensure that these interventions are effective, ethically sound, and patient-centered.
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CHARM and EvoETR: Precision epigenetic tools for gene silencing.
With the advent of gene editing technologies like CRISPR/Cas9, it has become possible to edit genomic regions of interest for research and therapeutic purposes. These technologies have also been adapted to alter gene expression without changing their DNA sequence, allowing epigenetic edits. While genetic editors make edits by cutting the genome at specified regions, epigenetic editors leverage the same targeting mechanism but act based on the epigenetic modifier fused to them, such as a methyltransferase. Here, we discuss two recently employed epigenetic editors (epi-editors) that silenced target genes involved in disease to mitigate their effects. Neumann et al. reported the construction of an epigenetic editor called CHARM that could methylate and silence the prion gene in mouse brains and subsequently switch itself off. Additionally, Capelluti et al. developed an epi-editor called EvoETR that knocked down Pcsk9 in the murine liver to reduce LDL levels. We aim to highlight the design principles underlying the design of these epi-editors to inform future editor designs.
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CRISP-ID: decoding CRISPR mediated indels by Sanger sequencing.
The advent of next generation gene editing technologies has revolutionized the fields of genome engineering in allowing the generation of gene knockout models and functional gene analysis. However, the screening of resultant clones remains challenging due to the simultaneous presence of different indels. Here, we present CRISP-ID, a web application which uses a unique algorithm for genotyping up to three alleles from a single Sanger sequencing trace, providing a robust and readily accessible platform to directly identify indels and significantly speed up the characterization of clones.
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Brave new dialogue.
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Global developments of genome editing in agriculture.
Genome editing, particularly using of site-directed nucleases such as the CRISPR system, has spread rapidly through the biological sciences. Genome editing in crops could significantly speed up the progress of breeding programs. It could drive the development of traits in new crops and allow improvements in yield and pest resistance, adaptation to climate change, and industrial and pharmaceutical applications. However biofortification is a key challenge to satisfy nutritional needs in vitamins for developing countries and new consumer's needs for developed countries. China and the USA lead scientific research in crop editing. Nigeria, being headquarters to numerous research consortia, is the most involved country in Africa. Genome editing in animals including pig, cattle, sheep, and carp, has not merely accelerated research but has made possible research that was previously unfeasible. It has been used to increase disease resistance, to make livestock better adapted to farming or environmental conditions, to increase fertility and growth, and to improve animal welfare. The USA, the UK and China are the most involved countries in animal genome editing. Global food production needs to increase as much as 70 per cent to support the growing population. Genome editing could contribute improving the efficiency of food distribution and reducing waste. Depending on the regulatory conditions, genome editing could open up the field to smaller companies and public labs.
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Engineering a compact genome-editing tool.
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Delivering genome editing tools to primary cells.
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Human germline editing needs one message.
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AAV-mediated gene editing lights up the lung.
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CRISPR still needs microbiologists.
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