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Discovery in CRISPR-Cas9 system.
The 2020 Nobel Prize in Chemistry was awarded to the American scientist Jennifer A. Doudna and the French scientist Emmanuelle Charpentier, in recognition of their discovery in one of the greatest weapons in genetic technology: CRISPR-Cas9 gene scissors. The CRISPR-Cas system is a bacterial defense immune system against exogenous genetic material. Because the system can specifically recognize and cut DNA, this technology is widely used for precise editing of animal, plant, and microbial DNA. The discovery of CRISPR-Cas9 gene scissors enables the tedious and complicated cell gene editing work to be completed in a few weeks or even less, which has promoted the development of gene editing technology in various fields and brought revolutionary influence to the field of life sciences. At the same time, CRISPR gene editing technology has become one of the new therapies for tumors because of its large number of targets and relatively simple operation, and it also makes gene therapy possible. Although the technology still needs to solve technical problems such as off-target and promoter inefficiency, the CRISPR-Cas system will show its unique advantages in more fields with the continuous development of life science and basic medicine.
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Targeted Human Editing Using CRISPR Technology in Neural Stem Cells.
Development of the programmable nuclease Ca9 has increased our understanding of the molecular basis of the physiological processes and diseases demonstrating a remarkable potential across various fields, including basic research, applied biotechnology, and biomedical research. CRISPR-Cas9 is a gene-editing technique that uses a guide RNA to direct the Cas9 enzyme to specific DNA sequences, where it creates double-strand breaks. These breaks can be repaired through either nonhomologous end joining (NHEJ), leading to gene mutations, or homology-directed repair (HDR), enabling precise edits. These characteristics render Cas9 an ideal candidate for various genome editing applications, particularly for the generation of knockout cells to study gene function either in vitro or in vivo. Delivery of the CRISPR molecular machinery is a crucial step for successful editing. Lentiviral vectors have been shown as an extremely effective method to transduce a wide range of cell types including neurons, astrocytes, adult neuronal stem cells, and oligodendrocytes. Here, we provide a useful protocol based on the lentiviral approach to develop CRISPR edition, facilitating the study of gene function in mammalian cells.
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Advancements and challenges in gene editing for improvement of vegetatively propagated crops.
Gene editing technologies, particularly CRISPR-Cas9, have revolutionized agriculture by offering precise and efficient tools to enhance crop production. The vegetatively propagated crops, crucial for global food security, face challenges such as climate change, pests, and limited genetic diversity. CRISPR-Cas9 enables targeted modifications to improve traits like disease resistance, drought tolerance, and nutritional content, thereby boosting productivity and sustainability. Despite its transformative potential, the adoption of gene editing in vegetatively propagated crops is hampered by technical complexities and regulatory frameworks. This review explores recent advancements, challenges, and prospects of gene editing in vegetatively propagated crops, emphasizing strategies to overcome technical barriers and regulatory constraints. Addressing these issues is essential for realizing the full agricultural potential of gene editing and ensuring food security in a changing global climate.
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The Delivery Challenge of Genome Editing in Human Epithelia.
Despite exciting advances in gene editing, their clinical translation is still hampered by the lack of delivery systems that can encapsulate and deliver gene editing tools like CRISPR-Cas9 or prime editors to the target side. This is particularly challenging in human epithelia, such as the skin and the lung; the latter of which being a mucosal surface that is covered by a mucus layer. In this perspective, the design and biological assessment of delivery systems for gene editing tools like CRISPR in skin and mucosal surfaces are discussed. The current state-of-the-art, current knowledge, and translational gaps, and guide toward improved translation are highlighted.
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Plasmid-Based CRISPR-Cas9 Editing in Multiple Candida Species.
CRISPR-Cas9 technology radically changed the approach to genetic manipulation of both medically and industrially relevant Candida species, as attested by the ever-increasing number of applications to the study of pathogenesis, drug resistance, gene expression, and host pathogen interaction and drug discovery. Here, we describe the use of plasmid-based systems for high efficiency CRISPR-Cas9 gene editing into any strain of four non-albicans Candida species, namely, Candida parapsilosis, Candida orthopsilosis, Candida metapsilosis, and Candida tropicalis. The plasmids pCP-tRNA and pCT-tRNA contain all the elements necessary for expressing the CRISPR-Cas9 machinery, and they can be used in combination with a repair template for disrupting gene function by insertion of a premature stop codon or by gene deletion. The plasmids are easily lost in the absence of selection, allowing scarless gene editing and minimizing detrimental effects of prolonged Cas9 expression.
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CRISPR-Cas9: New Heights, New Hesitations.
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A GCSFR/CSF3R zebrafish mutant models the persistent basal neutrophil deficiency of severe congenital neutropenia.
Granulocyte colony-stimulating factor (GCSF) and its receptor (GCSFR), also known as CSF3 and CSF3R, are required to maintain normal neutrophil numbers during basal and emergency granulopoiesis in humans, mice and zebrafish. Previous studies identified two zebrafish CSF3 ligands and a single CSF3 receptor. Transient antisense morpholino oligonucleotide knockdown of both these ligands and receptor reduces neutrophil numbers in zebrafish embryos, a technique widely used to evaluate neutrophil contributions to models of infection, inflammation and regeneration. We created an allelic series of zebrafish csf3r mutants by CRISPR/Cas9 mutagenesis targeting csf3r exon 2. Biallelic csf3r mutant embryos are viable and have normal early survival, despite a substantial reduction of their neutrophil population size, and normal macrophage abundance. Heterozygotes have a haploinsufficiency phenotype with an intermediate reduction in neutrophil numbers. csf3r mutants are viable as adults, with a 50% reduction in tissue neutrophil density and a substantial reduction in the number of myeloid cells in the kidney marrow. These csf3r mutants are a new animal model of human CSF3R-dependent congenital neutropenia. Furthermore, they will be valuable for studying the impact of neutrophil loss in the context of other zebrafish disease models by providing a genetically stable, persistent, reproducible neutrophil deficiency state throughout life.
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Gene Editing Applications as Future Cardiovascular Therapies.
Cardiovascular disease is the leading cause of global morbidity and mortality, despite advances in pharmacological and surgical interventions. The emergence of CRISPR-Cas9 genome editing technology offers promising approaches for correcting genetic causes of hereditary cardiovascular disorders and modulating pathogenic signaling pathways implicated in various heart diseases. However, several challenges with respect to in vivo delivery of gene editing components, as well as important safety considerations, remain to be addressed in the path toward possible clinical application. We review current gene editing strategies, their potential therapeutic applications in the context of a variety of cardiovascular disorders, and their respective merits, limitations, and regulatory considerations. The rapid advances in this field combined with the many opportunities for deploying gene editing therapies for cardiovascular disorders augur well for the future of this transformative technology.
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China tightens rules on gene editing.
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Combining Engineered Nucleases with Adeno-associated Viral Vectors for Therapeutic Gene Editing.
With the recent advent of several generations of targeted DNA nucleases, most recently CRISPR/Cas9, genome editing has become broadly accessible across the biomedical community. Importantly, the capacity of these nucleases to modify specific genomic loci associated with human disease could render new classes of genetic disease, including autosomal dominant or even idiopathic disease, accessible to gene therapy. In parallel, the emergence of adeno-associated virus (AAV) as a clinically important vector raises the possibility of integrating these two technologies towards the development of gene editing therapies. Though clear challenges exist, numerous proof-of-concept studies in preclinical models offer exciting promise for the future of gene therapy.
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