energia: Jennifer Doudna

Evidence: 100 • authority: 72

Jennifer Anne Doudna (; born February 19, 1964) is an American biochemist who has pioneered work in CRISPR gene editing, and made other fundamental contributions in biochemistry and genetics. She received the 2020 Nobel Prize in Chemistry, with Emmanuelle Charpentier, "for the development of a method for genome editing." She is the Li Ka Shing Chancellor's Chair Professor at the University of California, Berkeley. She has been an investigator with the Howard Hughes Medical Institute since 1997. In 2012, Doudna and Emmanuelle Charpentier were the first to propose that CRISPR-Cas9 (enzymes from bacteria that control microbial immunity) could be used for programmable editing of genomes, which…

Evidence: 68 • authority: 72

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 ("within the living"). The technique is considered highly significant in biotechnology and medicine as it enables in vivo genome editing and is considered exc…

Evidence: 68 • authority: 72

CRISPR (; acronym for clustered regularly interspaced short palindromic repeats) is a family of DNA sequences found in the genomes of prokaryotic organisms such as bacteria and archaea. Each sequence within an individual prokaryotic CRISPR is derived from a DNA fragment of a bacteriophage that had previously infected the prokaryote or one of its ancestors. These sequences are used to detect and destroy DNA from similar bacteriophages during subsequent infections. Hence these sequences play a key role in the antiviral (i.e. anti-phage) defense system of prokaryotes and provide a form of heritable, acquired immunity. CRISPR is found in approximately 50% of sequenced bacterial genomes and near…

Evidence: 60 • authority: 92

Editorial: First Regulatory Approvals for CRISPR-Cas9 Therapeutic Gene Editing for Sickle Cell Disease and Transfusion-Dependent β-Thalassemia.. In 2020, Emmanuelle Charpentier and Jennifer Doudna were awarded the Nobel Prize in Chemistry for their research on the endonuclease, clustered regularly interspaced short palindromic repeats (CRISPR) and the CRISPR-associated protein 9 (CRISPR-Cas9) method for DNA editing. On 16 November 2023, the UK Medicines and Healthcare Products Regulatory Agency (MHRA) was the first to approve the CRISPR-Cas9 gene editing therapy, Casgevy (exagamglogene autotemcel), for the treatment of patients with transfusion-dependent b-thalassemia and the treatment of s…

Evidence: 60 • authority: 72

Cas9 (CRISPR-associated protein 9) is a DNA cutting endonuclease that is part of the CRISPR immune system in bacteria and archaea. It has been adapted for use as a CRISPR genome editing tool. The CRISPR-Cas9 system is widely used in genetic engineering, medicine, agriculture, and biotechnology. The Cas9 endonuclease introduces site-specific double-stranded breaks in DNA, enabling gene inactivation, removal, or insertion through cellular repair mechanisms such as non-homologous end joining or homologous recombination. Cas9 was discovered in Streptococcus pyogenes and later in S. thermophilus. It functions with two small RNAs—crRNA and tracrRNA—to form a four-component complex. In 2012, Doudn…

Evidence: 52 • authority: 92

Emerging Gene-editing nano-therapeutics for Cancer. Remarkable progress has been made in the field of genome engineering after the discovery of CRISPR/Cas9 in 2012 by Jennifer Doudna and Emmanuelle Charpentier. Compared to any other gene-editing tools, CRISPR/Cas9 attracted the attention of the scientific community because of its simplicity, specificity, and multiplex editing possibilities for which the inventors were awarded the Nobel prize for chemistry in 2020. CRISPR/Cas9 allows targeted alteration of the genomic sequence, gene regulation, and epigenetic modifications using an RNA-guided site-specific endonuclease. Though the impact of CRISPR/Cas9 was undisputed, some of its limitations…

Evidence: 42 • authority: 72

Emmanuelle Marie Charpentier (French pronunciation: [emanɥɛl maʁi ʃaʁpɑ̃tje]; born 11 December 1968) is a French academic, microbiologist, geneticist, and biochemist. She has served as a director at the Max Planck Institute for Infection Biology in Berlin since 2015. Three years later, she founded an independent research institute, the Max Planck Unit for the Science of Pathogens. In 2020, Charpentier and American biochemist Jennifer Doudna of the University of California, Berkeley, were awarded the Nobel Prize in Chemistry "for the development of a method for genome editing" (through CRISPR). This was the first science Nobel Prize ever won exclusively by two women.

Evidence: 34 • authority: 92

CRISPR-Cas9-mediated therapeutics: Current clinical trials and therapy approval landscape to treat human diseases.. The genome editing technology using CRISPR-Cas9 has created a new trajectory for treating diverse human diseases. The approval of CRISPR-Cas9 therapeutics was first given to Casgevy for treating transfusion-dependent β-thalassemia (TDT) by the UK Medicines and Healthcare Products Regulatory Agency (UKMHRA) on November 16, 2023. Subsequently, UKMHRA approved Casgevy for the treatment of sickle cell disease (SCD), which was approved by the US Food and Drug Administration (FDA) on December 8, 2023. The approval has created a new era of gene editing medicine. Numerous clinical tri…

Evidence: 31 • authority: 88

The new frontier of genome engineering with CRISPR-Cas9. The advent of facile genome engineering using the bacterial RNA-guided CRISPR-Cas9 system in animals and plants is transforming biology. We review the history of CRISPR (clustered regularly interspaced palindromic repeat) biology from its initial discovery through the elucidation of the CRISPR-Cas9 enzyme mechanism, which has set the stage for remarkable developments using this technology to modify, regulate, or mark genomic loci in a wide variety of cells and organisms from all three domains of life. These results highlight a new era in which genomic manipulation is no longer a bottleneck to experiments, paving the way toward fundame…

Evidence: 25 • authority: 80

Jennifer A. Doudna and Emmanuelle Charpentier

Evidence: 25 • authority: 80

Emmanuelle Charpentier y Jennifer Doudna, premio Princesa de Asturias de Investigación Científica y Técnica 2015

Evidence: 25 • authority: 80

Emmanuelle Charpentier and Jennifer A. Doudna