From Laboratory to Fields: Unlocking the Potential of CRISPR-Driven Biopesticides

The Broad Institute Scores Another Victory in Its Battle with the University of California over the Patenting of CRIPSR

Application of the CRISPR–Cas System for Efficient Genome Engineering in Plants

British Society for Gene and Cell Therapy Annual Conference and Joint UK Regenerative Medicine Platform Meeting Royal Welsh College of Music & Drama Cardiff, Wales, United Kingdom Wednesday April 19-Friday April 21, 2017 Conference Abstracts.

CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) is a versatile technology that allows precise modification of genes. One of its most promising applications is in cancer treatment. By targeting and editing specific genes involved in cancer development and progression, CRISPR has the potential to become a powerful tool in the fight against cancer. This review aims to assess the recent progress in CRISPR technology for cancer research and to examine the obstacles and potential strategies to address them. The two most commonly used CRISPR systems for gene editing are CRISPR/Cas9 and CRISPR/Cas12a. CRISPR/Cas9 employs different repairing systems, including homologous recombination (HR) and nonhomologous end joining (NHEJ), to introduce precise modifications to the target genes. However, off-target effects and low editing efficiency are some of the main challenges associated with this technology. To overcome these issues, researchers are exploring new delivery methods and developing CRISPR/Cas systems with improved specificity. Moreover, there are ethical concerns surrounding using CRISPR in gene editing, including the potential for unintended consequences and the creation of genetically modified organisms. It is important to address these issues through rigorous testing and strict regulations. Despite these challenges, the potential benefits of CRISPR in cancer therapy cannot be overlooked. By introducing precise modifications to cancer cells, CRISPR could offer a targeted and effective treatment option for patients with different types of cancer. Further investigation and development of CRISPR technology are necessary to overcome the existing challenges and harness its full potential in cancer therapy.

The emergence of an inexpensive and efficient tool for gene editing is accelerating the pace of cardiovascular research and beginning to show potential as a treatment tool. The new gene-editing technology leverages a bacterial defense mechanism against viral infections. Bacteria store sections of viral DNA in repeated sequences called CRISPR within their own genomes and use those sequences to identify and destroy viruses. Now, scientists are inserting genes they would like to target into CRISPR and pairing it with CAS9, an enzyme that cuts DNA. This process allows scientists to efficiently remove or replace a targeted gene. “It’s been a transformative technology,” said Kiran Musunuru, MD, PhD, MPH, an associate professor of cardiovascular medicine and genetics at the University of Pennsylvania’s Perelman School of Medicine. Gene editing has been a common tool in research for decades—particularly for producing model organisms with a particular genetic mutation. However, older techniques were more time-consuming, expensive, and more error-prone. CRISPR-CAS is allowing scientists to more easily and quickly produce model animals or human cell lines with specific genetic variants allowing experiments to proceed at an unprecedented pace or scale. Some preliminary studies have suggested that it also may be a useful tool for treating some forms of heart disease. The most immediate impact of CRISPR-CAS9 has been speeding the production …

ABSTRACTGenome editing technologies, particularly clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9, have transformed biomedical research by enabling precise genetic modifications. Due to its efficiency, cost-effectiveness and versatility, CRISPR has been widely applied across various stages of research, from fundamental biological investigations in preclinical models to potential therapeutic interventions. In nephrology, CRISPR represents a groundbreaking tool for elucidating the molecular mechanisms underlying kidney diseases and developing innovative therapeutic approaches. This review synthesizes the latest advancements in CRISPR-based gene editing within nephrology, highlighting its applications in genetic kidney disorders, polygenic nephropathies and functional genomic studies. Preclinical studies utilizing CRISPR-engineered kidney organoids and animal models have provided crucial insights into disease pathophysiology, offering platforms for drug discovery and precision medicine. Additionally, CRISPR-based functional screens have identified novel disease-associated pathways, particularly in diabetic nephropathy and glomerular disorders. Beyond experimental research, the therapeutic potential of CRISPR in nephrology is emerging, with recent advances in base editing and prime editing demonstrating the feasibility of correcting pathogenic mutations in conditions such as Alport syndrome and autosomal dominant polycystic kidney disease. Moreover, CRISPR plays a pivotal role in xenotransplantation, with gene-edited porcine kidneys addressing key immunological and virological barriers. Despite its promise, clinical translation faces challenges, including delivery efficiency, off-target effects and ethical considerations. This review provides an overview of the current state and future directions of CRISPR-based gene editing in nephrology, underscoring its transformative potential in advancing kidney disease research and therapeutics.

COSMID: A Web-based Tool for Identifying and Validating CRISPR/Cas Off-target Sites.

CRISPR genetic toolkits of classical food microorganisms: Current state and future prospects

In It Together

Context: The clustered regularly interspaced short palindromic repeat (CRISPR)/Cas system is a groundbreaking gene-editing tool that shows great promise for modifying genomes. Derived from prokaryotic adaptive immune defense mechanisms, this technique has been used in research on human diseases, demonstrating remarkable therapeutic potential. Through CRISPR, specific genetic mutations in patients can be corrected during gene therapy, offering a solution for treating diseases that were previously untreatable using conventional methods. This review explores the recent progress and future prospects of the CRISPR system, focusing on its applications in medicine and stem cell engineering. Special emphasis is placed on medical applications, the latest target design or analysis tools for genome editing, advancements in stem cell engineering, and associated innovations and challenges. Evidence Acquisition: This study reviewed articles indexed in ISI, SID, PubMed, and PubMed Central from 2007 to 2024. Results: Cas9, a key protein in CRISPR gene editing, is an endonuclease capable of targeting and cutting specific DNA sequences, guided by short RNA sequences. The gene editing process involves homology-directed repair (HDR), non-homologous end joining (NHEJ), and base editing pathways. Base editing, which modifies the epigenome without inducing DNA breaks, is gaining increasing attention. However, CRISPR still faces technical challenges, and the development of more efficient "super" CRISPR technology will likely require time. This article reviews the effectiveness, limitations, and applications of the CRISPR system. Conclusions: CRISPR/Cas9 tools allow for the creation of precise models, leading to more effective treatment options for patients.

What Is the Issue? The first therapeutics based on clustered regularly interspaced short palindromic repeats (CRISPR) technologies are entering the market. These gene editing technologies have the potential to change treatment paradigms and may be used to treat conditions that cannot be treated or cured with current methods. This report aims to provide an overview of the technologies and their current and potential roles in health care. What Are the Technologies? CRISPR is a part of bacterial immune systems that can cut DNA strands and is used as a gene editing tool. A guide ribonucleic acid (RNA) sequence leads the CRISPR-associated nuclease to the target DNA sequence where the cut is made. These edits change the function of the gene, making genes nonfunctional or replacing the coding sequence for 1 gene with another. CRISPR can also be used to increase or decrease the expression of specific genes. What Is the Potential Impact? CRISPR-based technologies have a variety of potential applications in health care, including: treating genetic diseases understanding the genetic mechanisms of diseases and investigating the relevance of potential drug treatments managing infectious diseases through detection, treatment, and elimination. What Else Do We Need to Know? Ethical issues pertinent to the use of CRISPR include the ability to obtain adequately informed consent, the potential future consequences of gene editing and its potential unintended effects, and the impact gene editing could have on future generations. The long-term effects of CRISPR-based therapies are currently unknown. Further research into emerging applications is required. Long-term follow-up of the patients who have received the first CRISPR-based therapeutics will help inform understanding of the safety and effectiveness of these treatments. While the first of these therapies have been granted regulatory authorization, the next viable CRISPR-based therapies are still in the early phases of development, with the pivotal clinical trials not expected to be completed until at least 2027.

Gene editing is defined as creating targeted changes in the genome using sequence-specific nucleases. The CRISPR (clustered regularly interspaced short palindromic repeats)/Cas9 (CRISPR-associated protein 9) system provides a gene editing tool that has led to significant advances in this field and the development of potentially curative strategies for a variety of disorders. Most of the research on gene therapy and gene editing for muscle disorders has focused on Duchenne muscular dystrophy (DMD), a fatal, progressive X-linked neuromuscular disorder resulting from the absence of dystrophin. The molecular aspects of DMD present challenging obstacles to gene therapy; however, the versatility of the CRISPR system is providing ingenious ways of circumventing those obstacles. In this chapter, we review gene editing tools, notably zinc-finger nucleases, transcription activator-like effector nucleases, and CRISPR. We then discuss the uses of CRISPR in muscle disorders, focusing on DMD, as well as challenges inherent to gene editing of muscle cells.

At present, the main gene editing tools encompass TREN, Zinc Finger Nucleases (ZFN), clustered regularly interspaced short palindromic repeats (CRISPR), and Transcription Activator-Like Effector Nucleases (TALEN). In this study, we introduce an overview of the three gene editing methodologies and discuss their current clinical applications. In addition, we suggest some trends and future applications within the field of gene editing. ZFNs represent one of the pioneering technologies, demonstrating significant efficacy in mitigating a multitude of genetic diseases and finding applications in agriculture. Yet, this technology contains intricate processes and produces substantial costs when implemented. TALENs have already been employed across various domains. In the medical field, they have been successfully applied in the treatment of leukemia in infants. However, TALENs are being replaced by CRISPR due to the superior efficiency of CRISPR. CRISPR, consisting of six components, exhibits considerable promise in the medical realm, particularly in the context of treating diseases such as Alzheimer's disease (AD). In the realm of genetic engineering, it can collaborate with B cells to rectify specific genes within the human genome, which have been tested in experiments. In the future, it can be used in many fields, including agriculture and nucleic acid testing.

Clustered regularly interspaced short palindromic repeats (CRISPR) genome editing has already reinvented the direction of genetic and stem cell research. For more complex diseases it allows scientists to simultaneously create multiple genetic changes to a single cell. Technologies for correcting multiple mutations in an in vivo system are already in development. On the surface, the advent and use of gene editing technologies is a powerful tool to reduce human suffering by eradicating complex disease that has a genetic etiology. Gene drives are CRISPR mediated alterations to genes that allow them to be passed on to subsequent populations at rates that approach one hundred per cent transmission. Therefore, from an anticipatory biomedical ethics perspective, it is possible to conceive gene drive being used with CRISPR to permanently ameliorate aberrant genes from wild-type populations containing mutations. However, there are also a number of possible side effects that could develop as the result of combining gene editing and gene drive technologies in an effort to eradicate complex diseases. In this paper, we critically analyse the hypothesis that the combination of CRISPR and gene drive will have a deleterious effect on human populations from an ethical perspective by developing an anticipatory ethical analysis of the implications for the use of CRISPR together with gene drive in humans.

CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) and other gene-editing technologies have revolutionized genetic research by enabling precise, targeted modifications of DNA sequences. This paper provides a comprehensive exploration of CRISPR technology, detailing its development, mechanism of action, and versatility in diverse applications. From advancements in medicine, including therapeutic interventions for genetic disorders, to innovations in agriculture aimed at enhancing crop resilience and yield, CRISPR's transformative potential is vast. However, the rapid evolution of gene editing presents significant ethical and societal challenges, particularly concerning human germline editing, ecological impacts, and issues of accessibility and equity. This paper examines these ethical considerations, emphasizing the need for robust regulatory frameworks and responsible scientific practices. It also projects the future trajectory of gene editing technologies, speculating on emerging trends, possible breakthroughs, and the global implications of CRISPR in fields such as personalized medicine, synthetic biology, and biotechnology. By critically analyzing current applications and addressing ethical concerns, this study aims to provide a balanced perspective on CRISPR's potential to reshape genetic research while advocating for ethical governance and public engagement in its ongoing development. CRISPR’s ability to target specific genes with high accuracy has made it an invaluable tool not only in research laboratories but also in clinical settings, where it shows promise in treating previously incurable diseases. Recent advancements have extended CRISPR’s applications beyond simple gene knockout, allowing for base editing, prime editing, and epigenetic modifications that expand the possibilities for genetic correction and enhancement. As scientists explore using CRISPR in complex organisms, the precision and control required for safe and effective treatments become a key focus, particularly in addressing off-target effects that could lead to unintended genetic consequences.