LEAPER Gene Editing
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LEAPER Gene Editing
LEAPER (Leveraging endogenous ADAR for programmable editing of RNA) is a genetic engineering technique in molecular biology by which RNA can be edited. The technique relies on engineered strands of RNA to recruit native ADAR enzymes to swap out different compounds in RNA. Developed by researchers at Peking University in 2019, the technique, some have claimed, is more efficient than the CRISPR gene editing technique. Initial studies have claimed that editing efficiencies of up to 80%. Synopsis As opposed to DNA gene editing techniques (e.g., using CRISPR-Cas proteins to make modifications directly to a defective gene), LEAPER targets editing messenger RNA (mRNA) for the same gene which is transcribed into a protein. Post-transcriptional RNA modification typically involves the strategy of converting adenosine-to-inosine (A-to-I) since inosine (I) demonstrably mimics guanosine (G) during translation into a protein. A-to-I editing is catalyzed by adenosine deaminase acting on RNA (A ...
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Protein ADAR PDB 1qbj
Proteins are large biomolecules and macromolecules that comprise one or more long chains of amino acid residues. Proteins perform a vast array of functions within organisms, including catalysing metabolic reactions, DNA replication, responding to stimuli, providing structure to cells and organisms, and transporting molecules from one location to another. Proteins differ from one another primarily in their sequence of amino acids, which is dictated by the nucleotide sequence of their genes, and which usually results in protein folding into a specific 3D structure that determines its activity. A linear chain of amino acid residues is called a polypeptide. A protein contains at least one long polypeptide. Short polypeptides, containing less than 20–30 residues, are rarely considered to be proteins and are commonly called peptides. The individual amino acid residues are bonded together by peptide bonds and adjacent amino acid residues. The sequence of amino acid residues in ...
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Oligonucleotide
Oligonucleotides are short DNA or RNA molecules, oligomers, that have a wide range of applications in genetic testing, research, and forensics. Commonly made in the laboratory by solid-phase chemical synthesis, these small bits of nucleic acids can be manufactured as single-stranded molecules with any user-specified sequence, and so are vital for artificial gene synthesis, polymerase chain reaction (PCR), DNA sequencing, molecular cloning and as molecular probes. In nature, oligonucleotides are usually found as small RNA molecules that function in the regulation of gene expression (e.g. microRNA), or are degradation intermediates derived from the breakdown of larger nucleic acid molecules. Oligonucleotides are characterized by the sequence of nucleotide residues that make up the entire molecule. The length of the oligonucleotide is usually denoted by " -mer" (from Greek ''meros'', "part"). For example, an oligonucleotide of six nucleotides (nt) is a hexamer, while one of 25 nt wou ...
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Genome Editing
Genome editing, or genome engineering, or gene editing, is a type of genetic engineering in which DNA is inserted, deleted, modified or replaced in the genome of a living organism. Unlike early genetic engineering techniques that randomly inserts genetic material into a host genome, genome editing targets the insertions to site-specific locations. The basic mechanism involved in genetic manipulations through programmable nucleases is the recognition of target genomic loci and binding of effector DNA-binding domain (DBD), double-strand breaks (DSBs) in target DNA by the restriction endonucleases ( FokI and Cas), and the repair of DSBs through homology-directed recombination (HDR) or non-homologous end joining (NHEJ). History Genome editing was pioneered in the 1990s, before the advent of the common current nuclease-based gene editing platforms, however, its use was limited by low efficiencies of editing. Genome editing with engineered nucleases, i.e. all three major classes of ...
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Genetic Engineering
Genetic engineering, also called genetic modification or genetic manipulation, is the modification and manipulation of an organism's genes using technology. It is a set of technologies used to change the genetic makeup of cells, including the transfer of genes within and across species boundaries to produce improved or novel organisms. New DNA is obtained by either isolating and copying the genetic material of interest using recombinant DNA methods or by artificially synthesising the DNA. A construct is usually created and used to insert this DNA into the host organism. The first recombinant DNA molecule was made by Paul Berg in 1972 by combining DNA from the monkey virus SV40 with the lambda virus. As well as inserting genes, the process can be used to remove, or "knock out", genes. The new DNA can be inserted randomly, or targeted to a specific part of the genome. An organism that is generated through genetic engineering is considered to be genetically modified (GM) an ...
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Prime Editing
Prime editing is a 'search-and-replace' genome editing technology in molecular biology by which the genome of living organisms may be modified. The technology directly writes new genetic information into a targeted DNA site. It uses a fusion protein, consisting of a catalytically impaired Cas9 endonuclease fused to an engineered reverse transcriptase enzyme, and a prime editing guide RNA (pegRNA), capable of identifying the target site and providing the new genetic information to replace the target DNA nucleotides. It mediates targeted Insertion (genetics), insertions, Deletion (genetics), deletions, and base-to-base conversions without the need for double strand breaks (DSBs) or donor DNA templates. The technology is an early-stage, experimental genome editing method that has received mainstream press attention due to its potential uses in medical genetics. It utilizes methodologies similar to precursor genome editing technologies, including CRISPR/Cas9-mediated genome editing, CRIS ...
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NgAgo
NgAgo is a single-stranded DNA (ssDNA)-guided Argonaute endonuclease, an acronym for ''Natronobacterium'' ''gregoryi'' Argonaute. NgAgo binds 5′ phosphorylated ssDNA of ~24 nucleotides (gDNA) to guide it to its target site and will make DNA double-strand breaks at the gDNA site. Like the CRISPR/Cas system, NgAgo was reported by Chunyu Han et al. to be suitable for genome editing, but this has not been replicated. In contrast to Cas9, the NgAgo–gDNA system does not require a protospacer adjacent motif (PAM). Role NgAgo was proposed to be useful for genome editing in May 2016 because of the system’s high accuracy and efficiency, which was said to minimize off-target effects. The specificity of the gDNA is essential, as cleavage efficiency is impaired by a single nucleotide mismatch between the guide and target molecules. Using 5’ phosphorylated ssDNAs as guide molecules reduces the possibility of cellular oligonucleotides misleading NgAgo. A guide molecule can only be a ...
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Gene Knockout
A gene knockout (abbreviation: KO) is a genetic technique in which one of an organism's genes is made inoperative ("knocked out" of the organism). However, KO can also refer to the gene that is knocked out or the organism that carries the gene knockout. Knockout organisms or simply knockouts are used to study gene function, usually by investigating the effect of gene loss. Researchers draw inferences from the difference between the knockout organism and normal individuals. The KO technique is essentially the opposite of a gene knock-in. Knocking out two genes simultaneously in an organism is known as a double knockout (DKO). Similarly the terms triple knockout (TKO) and quadruple knockouts (QKO) are used to describe three or four knocked out genes, respectively. However, one needs to distinguish between heterozygous and homozygous KOs. In the former, only one of two gene copies (alleles) is knocked out, in the latter both are knocked out. Methods Knockouts are accomplished throu ...
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Bioethics
Bioethics is both a field of study and professional practice, interested in ethical issues related to health (primarily focused on the human, but also increasingly includes animal ethics), including those emerging from advances in biology, medicine and technologies. It proposes the discussion about moral discernment in society (what decisions are "good" or "bad" and why) and it is often related to medical policy and practice, but also to broader questions as environment, well-being and public health. Bioethics is concerned with the ethical questions that arise in the relationships among life sciences, biotechnology, medicine, politics, law, theology and philosophy. It includes the study of values relating to primary care, other branches of medicine ( "the ethics of the ordinary"), ethical education in science, animal, and environmental ethics, and public health. Etymology The term ''Bioethics'' (Greek , life; , behavior) was coined in 1927 by Fritz Jahr in an article about a "b ...
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Hurler Syndrome
Hurler syndrome, also known as mucopolysaccharidosis Type IH (MPS-IH), Hurler's disease, and formerly gargoylism, is a genetic disorder that results in the buildup of large sugar molecules called glycosaminoglycans (GAGs) in lysosomes. The inability to break down these molecules results in a wide variety of symptoms caused by damage to several different organ systems, including but not limited to the nervous system, skeletal system, eyes, and heart. The underlying mechanism is a deficiency of alpha-L iduronidase, an enzyme responsible for breaking down GAGs. Without this enzyme, a buildup of dermatan sulfate and heparan sulfate occurs in the body. Symptoms appear during childhood, and early death usually occurs. Other, less severe forms of MPS Type I include Hurler-Scheie Syndrome (MPS-IHS) and Scheie Syndrome (MPS-IS). Hurler syndrome is classified as a lysosomal storage disease. It is clinically related to Hunter syndrome (MPS II); however, Hunter syndrome is X-linked, wh ...
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Genetic Engineering
Genetic engineering, also called genetic modification or genetic manipulation, is the modification and manipulation of an organism's genes using technology. It is a set of technologies used to change the genetic makeup of cells, including the transfer of genes within and across species boundaries to produce improved or novel organisms. New DNA is obtained by either isolating and copying the genetic material of interest using recombinant DNA methods or by artificially synthesising the DNA. A construct is usually created and used to insert this DNA into the host organism. The first recombinant DNA molecule was made by Paul Berg in 1972 by combining DNA from the monkey virus SV40 with the lambda virus. As well as inserting genes, the process can be used to remove, or "knock out", genes. The new DNA can be inserted randomly, or targeted to a specific part of the genome. An organism that is generated through genetic engineering is considered to be genetically modified (GM) an ...
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Nature Biotechnology
''Nature Biotechnology'' is a monthly peer-reviewed scientific journal published by Nature Portfolio. The chief editor heads an in-house team of editors. The focus of the journal is biotechnology including research results and the commercial business sector of this field. Coverage includes the related biological, biomedical, agricultural and environmental sciences. Also of interest are the commercial, political, legal, and societal influences that affect this field. The journal continues serial publication of the title "Bio/Technology", which had a publication period of 1983 to 1996. Abstracting and indexing This journal is indexed in Catalog @ NPG
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Beijing
} Beijing ( ; ; ), alternatively romanized as Peking ( ), is the capital of the People's Republic of China. It is the center of power and development of the country. Beijing is the world's most populous national capital city, with over 21 million residents. It has an administrative area of , the third in the country after Guangzhou and Shanghai. It is located in Northern China, and is governed as a municipality under the direct administration of the State Council with 16 urban, suburban, and rural districts.Figures based on 2006 statistics published in 2007 National Statistical Yearbook of China and available online at archive. Retrieved 21 April 2009. Beijing is mostly surrounded by Hebei Province with the exception of neighboring Tianjin to the southeast; together, the three divisions form the Jingjinji megalopolis and the national capital region of China. Beijing is a global city and one of the world's leading centres for culture, diplomacy, politics, finance, busi ...
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