Gene editing

Extracted Attributes

• Mechanism

  Molecular scissors (removes/adds genes by cutting DNA at desired locations)

• Advantages

  Precise, cost-effective, efficient

• Applications

  New medicines, agricultural products, genetically modified organisms, treating genetic diseases, cancers, controlling pathogens and pests

• AI Model Type

  Protein language model (for OpenCrisper-1)

• AI-driven Tool

  OpenCrisper-1

• Key Technology

  CRISPR-Cas9 system

• Origin of Cas9

  Streptococcus pyogenes (bacterial species)

• Core Components

  Cas9 nuclease complexed with synthetic guide RNA (gRNA)

• Knock-in Mutation Method

  Homology Directed Repair (HDR)

• Knock-out Mutation Method

  Non-Homologous End Joining (NHEJ) or POLQ/polymerase theta-mediated end-joining (TMEJ)

• Newer Gene Editing Approach

  Base editing

Timeline

• The idea of using gene editing to treat disease or alter traits dates to at least this decade, following the discovery of DNA's double-helix structure. (Source: Web Search)

  1950s

• Genome editing in eukaryotic cells became possible using various methods, though these were often inefficient and impractical on a large scale. (Source: Wikipedia)

  1980s

• The CRISPR-Cas9-gRNA complex for genome editing was chosen as the AAAS's Breakthrough of the Year. (Source: Wikipedia)

  2015

• Jennifer Doudna and Emmanuelle Charpentier were awarded the Nobel Prize in Chemistry for their development of the CRISPR-Cas9 gene editing technique. (Source: Summary, Wikipedia)

  2020

• Casgevy, the first drug utilizing CRISPR gene editing, was approved for use in the United Kingdom for treating sickle-cell disease and beta thalassemia. (Source: Summary, Wikipedia)

  2023

• The Kingdom of Bahrain became the second country to approve Casgevy for treating sickle-cell anemia and beta thalassemia. (Source: Wikipedia)

  2023-12-02

• Casgevy was approved for use in the United States by the Food and Drug Administration (FDA). (Source: Wikipedia)

  2023-12-08

CRISPR gene editing

CRISPR gene editing (; pronounced like "crisper"; an abbreviation for "clustered regularly interspaced short palindromic repeats") is a genetic engineering technique in molecular biology by which the genomes of living organisms may be modified. It is based on a simplified version of the bacterial CRISPR-Cas9 antiviral defense system. By delivering the Cas9 nuclease complexed with a synthetic guide RNA (gRNA) into a cell, the cell's genome can be cut at a desired location, allowing existing genes to be removed or new ones added in vivo. The technique is considered highly significant in biotechnology and medicine as it enables editing genomes in vivo and is precise, cost-effective, and efficient. It can be used in the creation of new medicines, agricultural products, and genetically modified organisms, or as a means of controlling pathogens and pests. It also offers potential in the treatment of inherited genetic diseases as well as diseases arising from somatic mutations such as cancer. However, its use in human germline genetic modification is highly controversial. The development of this technique earned Jennifer Doudna and Emmanuelle Charpentier the Nobel Prize in Chemistry in 2020. The third researcher group that shared the Kavli Prize for the same discovery, led by Virginijus Šikšnys, was not awarded the Nobel prize. Working like genetic scissors, the Cas9 nuclease opens both strands of the targeted sequence of DNA to introduce the modification by one of two methods. Knock-in mutations, facilitated via homology directed repair (HDR), is the traditional pathway of targeted genomic editing approaches. This allows for the introduction of targeted DNA damage and repair. HDR employs the use of similar DNA sequences to drive the repair of the break via the incorporation of exogenous DNA to function as the repair template. This method relies on the periodic and isolated occurrence of DNA damage at the target site in order for the repair to commence. Knock-out mutations caused by CRISPR-Cas9 result from the repair of the double-stranded break by means of non-homologous end joining (NHEJ) or POLQ/polymerase theta-mediated end-joining (TMEJ). These end-joining pathways can often result in random deletions or insertions at the repair site, which may disrupt or alter gene functionality. Therefore, genomic engineering by CRISPR-Cas9 gives researchers the ability to generate targeted random gene disruption. While genome editing in eukaryotic cells has been possible using various methods since the 1980s, the methods employed had proven to be inefficient and impractical to implement on a large scale. With the discovery of CRISPR and specifically the Cas9 nuclease molecule, efficient and highly selective editing became possible. Cas9 derived from the bacterial species Streptococcus pyogenes has facilitated targeted genomic modification in eukaryotic cells by allowing for a reliable method of creating a targeted break at a specific location as designated by the crRNA and tracrRNA guide strands. Researchers can insert Cas9 and template RNA with ease in order to silence or cause point mutations at specific loci. This has proven invaluable for quick and efficient mapping of genomic models and biological processes associated with various genes in a variety of eukaryotes. Newly engineered variants of the Cas9 nuclease that significantly reduce off-target activity have been developed. CRISPR-Cas9 genome editing techniques have many potential applications. The use of the CRISPR-Cas9-gRNA complex for genome editing was the AAAS's choice for Breakthrough of the Year in 2015. Many bioethical concerns have been raised about the prospect of using CRISPR for germline editing, especially in human embryos. In 2023, the first drug making use of CRISPR gene editing, Casgevy, was approved for use in the United Kingdom, to cure sickle-cell disease and beta thalassemia.. On 2 December 2023, the Kingdom of Bahrain became the second country in the world to approve the use of Casgevy, to treat sickle-cell anemia and beta thalassemia. Casgevy was approved for use in the United States on December 8, 2023, by the Food and Drug Administration.

Web Search Results

• What are genome editing and CRISPR-Cas9?: MedlinePlus Genetics

  Genome editing (also called gene editing) is a group of technologies that give scientists the ability to change an organism's DNA. These technologies allow genetic material to be added, removed, or altered at particular locations in the genome. Several approaches to genome editing have been developed. A well-known one is called CRISPR-Cas9, which is short for clustered regularly interspaced short palindromic repeats and CRISPR-associated protein 9. The CRISPR-Cas9 system has generated a lot of [...] Genome editing is of great interest in the prevention and treatment of human diseases. Currently, genome editing is used in cells and animal models in research labs to understand diseases. Scientists are still working to determine whether this approach is safe and effective for use in people. It is being explored in research and clinical trials for a wide variety of diseases, including single-gene disorders such as cystic fibrosis, hemophilia, and sickle cell disease. It also holds promise for

• Gene Editing | - Gene & Cell Therapy Education

  Image 18_1.aspx?width=350&height=350)Simply put, gene editing is a type of gene therapy. They both target the cause of disease, such as a variant or mutation in a gene, by using genetic material to treat or prevent disease. Most gene therapy genes are delivered. When the vector carrying the working gene enters the cell it provides new instructions to produce either more or less of a certain protein depending on what is needed. For the body to function properly it is essential that the right [...] Gene editing has gained a lot of attention over the past few years, but that may leave you wondering: what exactly is it? Gene editing aims to be a one-time therapy that directly edits pieces of DNA within the cell. It's considered a type of gene therapy, which is the use of genetic material to treat or prevent disease. What’s the Difference: Gene Therapy vs. Gene Editing [...] Like any gene therapy, gene editing aims to be a one-time treatment with lasting positive effects that slow or stop disease progression for a lifetime. However, there is no guarantee, and this change is permanent. We are still very early in the development of gene editing and the process is far from complete. While gene editing therapies are still somewhat within their infancy stages the development that we have seen over the past few years promises a future that looks very bright for hundreds

• Gene Editing Explained - Verve Therapeutics

  Gene editing has demonstrated incredible potential in driving new therapeutic breakthroughs to treat disease and continues to evolve. Gene editing works by making a permanent change in a target gene, disrupting the production of certain proteins that cause an underlying disease. Two common forms of gene editing are CRISPR-Cas9 and base editing. [...] Newer gene editing approaches – such as base editing – can potentially address these limitations. ### What is Base Editing? Base editing is a next-generation form of gene editing. Base editors can most simply be compared to pencils, in their ability to "erase" and rewrite a specific letter in a gene. [...] Our investigational base editing technology is a form of gene editing that enables precise, one-time changes to DNA, potentially turning off genes that contribute to dangerously high cholesterol levels and increase a person's risk of heart attack or stroke. DELIVERY SYSTEM ### Learn more about delivery systems in gene editing, including our proprietary GalNAc-LNP system.

• Gene editing | Definition, History, & CRISPR-Cas9 - Britannica

  gene editing, the ability to make highly specific changes in the DNA sequence of a living organism, essentially customizing its genetic makeup. Gene editing is performed using enzymes, particularly nucleases that have been engineered to target a specific DNA sequence, where they introduce cuts into the DNA strands, enabling the removal of existing DNA and the insertion of replacement DNA. Key among gene-editing technologies is a molecular tool known as CRISPR-Cas9, a powerful technology [...] The idea of using gene editing to treat disease or alter traits dates to at least the 1950s and the discovery of the double-helix structure of DNA. In the mid-20th-century era of genetic discovery, researchers realized that the sequence of bases in DNA is passed (mostly) faithfully from parent to offspring and that small changes in the sequence can mean the difference between health and disease. Recognition of the latter led to the inescapable conjecture that with the identification of [...] The development of gene-editing technology for gene therapy, however, proved difficult. Much early progress focused not on correcting genetic mistakes in the DNA but rather on attempting to minimize their consequence by providing a functional copy of the mutated gene, either inserted into the genome or maintained as an extrachromosomal unit (outside the genome). While that approach was effective for some conditions, it was complicated and limited in scope.

• Human genome editing - World Health Organization (WHO)

  Genome editing is a method for making specific changes to the DNA of a cell or organism. It can be used to add, remove or alter DNA in the genome. Human genome editing technologies can be used on somatic cells (non-heritable), germline cells (not for reproduction) and germline cells (for reproduction).

• Instance Of

  Q4167410