#genome editing

[cbirt]

Through the advent of CRISPR-Cas12, scientists have managed to unlock a new world of gene editing and reprogramming, with important applications for diagnostics, therapeutics, agricultural science, and more. However, their bacterial origin may make them difficult to work with as researchers begin to explore the potential of human genome editing. A new study led by researchers from MIT found thousands of new RNA-guided enzymes known as Fanzors prevalent among eukaryotic organisms that show great promise as novel tools for mammalian genome editing.

RNA-programmable DNA nucleases are essential for the function of prokaryotes in the defense and proliferation of mobile elements. Such nucleases include the likes of CRISPR argonauts, as well as the nucleases that comprise the OMEGA systems (Obligate Mobile Element Guided Activity), which includes nucleases such as IscB, IsrB, and IshB, among others. Of these, one nuclease, the TnpB gene, contains a nuclease domain similar to the one found in the Cas12 gene, suggesting an evolutionary relationship between the two. This is further backed up by phylogenetic analysis conducted on the two domains, which indicates that different subtypes of Cas12 originated independently from different groups of TnpB enzymes. Furthermore, biochemical and cellular experiments have demonstrated that the TnpB-Omegas complex is an RNA-guided and programmable DNA endonuclease.

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[bpod-bpod]

Editing the Future

Jennifer Doudna – born on this day (19th February)– shared the 2020 Nobel Prize for Chemistry with Emmanuelle Charpentier for developing a genetic engineering technique called CRISPR-Cas9. Based on a naturally occurring defence system used by bacteria to expunge foreign DNA from their genome, CRISPR-Cas9 has revolutionised both biomedical and plant research readily revealing the impact of editing genes in living cells and model organisms, and is being applied in human genome editing to correct disease-causing gene faults and deliver gene therapies

Image by Christopher Michel, on Flickr

Image originally published with a Creative Commons Attribution 2.0 Generic (CC BY 2.0)

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[cyb3rbr4tzz]

"Artist genes" and the belonging enzymes have been created using the above mentiond tools and inserted into Lieuwe van Gogh's genome in order to elevate natural dispositions of creativity.

Sugababe, 2014-2021

by diemut strebe

replica of Vincent van Gogh’s ear, via mtDNA genome of great-great-great grandchild of Vincent’s mother

living genetically engineered, reprogrammed and immortalized chondrocytes seeded on a biodegradable scaffold, plasma, acrylic containers, pumpsystem, microphone, speakers

[industryupdate]

The global genome editing market was valued at USD 6.9 billion in 2024 and is expected to grow at a rate of over 19.6%, reaching USD 20.4 billion by 2030, as reported by P&S Intelligence.

[tritonmarketresearchamey]

Latest Advances in Gene and Cell Therapies Transform Healthcare

Gene and cell therapies represent a ground-breaking advancement in medical science, offering potential cures for a variety of previously untreatable diseases. These therapies are revolutionizing how we provide targeted healthcare by modifying genetic material or using cells to restore or alter biological functions. Early interventions in congenital disorders can significantly reduce long-term health complications, offering a healthier start to life for newborns. Thus, the potential of gene and cell therapies to transform medical treatments is immense, especially in the field of natal and prenatal care.

A notable example of gene therapy involved the birth of the first babies with edited genes. In 2018, Dr. Jiankui announced the birth of twin girls whose genes were edited using CRISPR technology. He edited and deactivated a gene known as CCR5 with the goal of conferring resistance to HIV in those girls.

Latest Developments in Gene and Cell Therapies

The field of gene and cell therapies is crucial in the mainstream as drug-regulating authorities approve treatments for diseases like lymphoma and muscular dystrophy. Let us explore the latest developments regarding these therapies.

Non-Hodgkin lymphoma (NHL) accounts for about 4% of all cancers in the US, with an estimated 80,620 new cases expected this year. In this regard, Bristol Myers Squibb’s Breyanzi, a CAR T cell therapy, was approved in 2024 by the FDA, which utilizes the patient’s immune system to target and destroy cancer cells.

In 2024, the FDA approved Sarepta Therapeutics’ Elevidys, a gene therapy for Duchenne muscular dystrophy (DMD), which affects approximately 1 in 3,500 to 5000 male births worldwide, typically manifesting between ages 3 and 6. This groundbreaking offers new hope by addressing the root cause of this debilitating condition.

Exploring Current and Future Applications

CRISPR and Genome Editing: CRISPR technology has revolutionized genome editing, offering precise modifications to DNA and correcting genetic defects at their source. This technology is being explored for a variety of applications including current and future applications. However, acquiring approvals to run trials on humans has always been challenging, yet the CTX001 stands out with its success in this regard. The CTX001 is an autologous gene-edited stem cell therapy developed by CRISPR Therapeutics and Vertex Pharmaceuticals.

Dr. Haydar Frangoul, the medical director at HCA Sarah Cannon Research Institute Center, has been treating the first patient in the CTX001 trial for SCD therapy. The patient had battled sickle cell disease for 34 years before undergoing this one-time treatment. Post-treatment, her blood showed a significant proportion of fetal hemoglobin levels, enabling her to avoid blood transfusions and pain attacks without major side effects.

Stem Cell Research: These cells have the unique ability to differentiate into various cell types, making them invaluable for regenerative medicine. Research in stem cell therapy aims to treat conditions such as Parkinson’s disease, diabetes, and spinal cord injuries by replacing damaged cells with healthy ones in the near future. A notable example is a study using device-encapsulated pancreatic precursor cells derived from human embryonic stem cells. This study has shown that increased cell doses in optimized devices lead to detectable insulin production and improved glucose control.

CAR-T Cell Therapy: This therapy has shown impressive results in treating certain types of leukemia and lymphoma, offering hope for patients who have not responded to traditional treatments. This innovative approach uses modified T-cells to target and kill cancer cells. The future of CAR-T therapy looks promising, thereby expanding its application to treat more types of cancers, including solid tumors.

Gene Silencing and RNA-based Therapies: Emerging technologies like RNA interference (RNAi) and antisense oligonucleotides (ASOs) are being developed to silence harmful genes. An RNAi therapy like ‘AMVUTTRA’ developed by Alnylam, is approved in the US for treating polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. Thus, the future use of RNA therapies includes the treatment of neurodegenerative diseases like Huntington’s disease.

Understanding Ethical Considerations & the Role of Regulatory Bodies

Ethical frameworks must evolve amidst the concerns regarding ‘designer babies’, where genetic modifications used to select desired traits pose significant ethical dilemmas. A prominent example is the controversy of using CRISPR technology in human embryos, who claimed to have created the first gene-edited babies, sparking ethical debates and leading to his imprisonment. Several studies emphasize the importance of international regulatory standards and effective governance to ensure the responsible use of gene editing technologies.

Amidst the rapid pace of technological advancement, regulating gene and cell therapies needs rigorous safety standards. The regulatory bodies and agencies like the FDA’s Center for Biologics Evaluation and Research (CBER) in the US and the European Medicines Agency (EMA) in the EU play a critical role. Their frameworks include guidelines for approval of regenerative medicines and conditional or time-limited authorizations to facilitate quicker access to innovative treatments.

What the future beholds?

The future of gene and cell therapies lies in their integration into personalized medicine based on the genetic makeup of individual patients. Companies like CRISPR Therapeutics, Editas Medicine, and Intellia Therapeutics are at the forefront of research, developing therapies that could revolutionize the treatment of genetic disorders. As these therapies become more refined and accessible, they could significantly extend healthy life spans and improve the quality of life for millions.

[sathiyario05-blog]

What is mean Cybersecurity??How its used for IT industries.

  One of the biggest and economically high value compare to other industries is Information Technology industries. Mostly service Based IT industries running in India.IT industries used Cybersecurity for protection. What is mean by Cybersecurity?? Cybersecurity is the Practice of Protecting systems,networks and Programs from digital attacks. Main role of Cybersecurity Protecting an…

[gilberthbolanos]

An Introduction to Legal, Regulatory and Intellectual Property Rights Issues in Biotechnology

The book “An Introduction to Legal, Regulatory and Intellectual Property Rights Issues in Biotechnology” covers a multitude of themes and some of the most important legal issues arising in relation to biotechnology, including the historical development of a legal framework sufficient to protect public safety (Chapter 1), the current biotechnology regulatory system and the rules directing the…

[ryan-sometimes]

Being a biochemistry student is so funny because I’ll be staring off into space and look deep in thought but really I’m just thinking about CRISPR. The Roman Empire of every biochem student

[tremendousnightmaregalaxy]

Watch "Gene Silencing by microRNAs" on YouTube

microRNA is the tool used to give instructions to the Messenger (mRNA) by silencing, editing altering, manipulating, or best yet to hijack your foundational biological system that keeps ur fatal organs from becoming fatally expired. and in wrong hands (inexp or malevolent) alike could, would, and only a matter of time will become detrimental to our way of life. Mark my words.

[jcmarchi]

Killing the messenger

New Post has been published on https://thedigitalinsider.com/killing-the-messenger/

Killing the messenger

Like humans and other complex multicellular organisms, single-celled bacteria can fall ill and fight off viral infections. A bacterial virus is caused by a bacteriophage, or, more simply, phage, which is one of the most ubiquitous life forms on earth. Phages and bacteria are engaged in a constant battle, the virus attempting to circumvent the bacteria’s defenses, and the bacteria racing to find new ways to protect itself.

These anti-phage defense systems are carefully controlled, and prudently managed — dormant, but always poised to strike.

New open-access research recently published in Nature from the Laub Lab in the Department of Biology at MIT has characterized an anti-phage defense system in bacteria, CmdTAC. CmdTAC prevents viral infection by altering the single-stranded genetic code used to produce proteins, messenger RNA.

This defense system detects phage infection at a stage when the viral phage has already commandeered the host’s machinery for its own purposes. In the face of annihilation, the ill-fated bacterium activates a defense system that will halt translation, preventing the creation of new proteins and aborting the infection — but dooming itself in the process.

“When bacteria are in a group, they’re kind of like a multicellular organism that is not connected to one another. It’s an evolutionarily beneficial strategy for one cell to kill itself to save another identical cell,” says Christopher Vassallo, a postdoc and co-author of the study. “You could say it’s like self-sacrifice: One cell dies to protect the other cells.”

The enzyme responsible for altering the mRNA is called an ADP-ribosyltransferase. Researchers have characterized hundreds of these enzymes — although a few are known to target DNA or RNA, all but a handful target proteins. This is the first time these enzymes have been characterized targeting mRNA within cells.

Expanding understanding of anti-phage defense

Co-first author and graduate student Christopher Doering notes that it is only within the last decade or so that researchers have begun to appreciate the breadth of diversity and complexity of anti-phage defense systems. For example, CRISPR gene editing, a technique used in everything from medicine to agriculture, is rooted in research on the bacterial CRISPR-Cas9 anti-phage defense system.

CmdTAC is a subset of a widespread anti-phage defense mechanism called a toxin-antitoxin system. A TA system is just that: a toxin capable of killing or altering the cell’s processes rendered inert by an associated antitoxin.

Although these TA systems can be identified — if the toxin is expressed by itself, it kills or inhibits the growth of the cell; if the toxin and antitoxin are expressed together, the toxin is neutralized — characterizing the cascade of circumstances that activates these systems requires extensive effort. In recent years, however, many TA systems have been shown to serve as anti-phage defense.

Two general questions need to be answered to understand a viral defense system: How do bacteria detect an infection, and how do they respond?

Detecting infection

CmdTAC is a TA system with an additional element, and the three components generally exist in a stable complex: the toxic CmdT, the antitoxin CmdA, and an additional component called a chaperone, CmdC.

If the phage’s protective capsid protein is present, CmdC disassociates from CmdT and CmdA and interacts with the phage capsid protein instead. In the model outlined in the paper, the chaperone CmdC is, therefore, the sensor of the system, responsible for recognizing when an infection is occurring. Structural proteins, such as the capsid that protects the phage genome, are a common trigger because they’re abundant and essential to the phage.

The uncoupling of CmdC exposes the neutralizing antitoxin CmdA to be degraded, which releases the toxin CmdT to do its lethal work. 

Toxicity on the loose

The researchers were guided by computational tools, so they knew that CmdT was likely an ADP-ribosyltransferase due to its similarities to other such enzymes. As the name suggests, the enzyme transfers an ADP ribose onto its target.

To determine if CmdT interacted with any sequences or positions in particular, they tested a mix of short sequences of single-stranded RNA. RNA has four bases: A, U, G, and C, and the evidence points to the enzyme recognizing GA sequences.

The CmdT modification of GA sequences in mRNA blocks their translation. The cessation of creating new proteins aborts the infection, preventing the phage from spreading beyond the host to infect other bacteria.

“Not only is it a new type of bacterial immune system, but the enzyme involved does something that’s never been seen before: the ADP-ribsolyation of mRNA,” Vassallo says.

Although the paper outlines the broad strokes of the anti-phage defense system, it’s unclear how CmdC interacts with the capsid protein, and how the chemical modification of GA sequences prevents translation.

Beyond bacteria

More broadly, exploring anti-phage defense aligns with the Laub Lab’s overall goal of understanding how bacteria function and evolve, but these results may have broader implications beyond bacteria.

Senior author Michael Laub, Salvador E. Luria Professor and Howard Hughes Medical Institute Investigator, says the ADP-ribosyltransferase has homologs in eukaryotes, including human cells. They are not well studied, and not among the Laub Lab’s research topics, but they are known to be up-regulated in response to viral infection.

“There are so many different — and cool — mechanisms by which organisms defend themselves against viral infection,” Laub says. “The notion that there may be some commonality between how bacteria defend themselves and how humans defend themselves is a tantalizing possibility.” 

[reports2030]

Genome Editing Market Industry Trends and Forecast to 2032

The Global Genome Editing Market report covers deep insights of various vital aspects of the market. Moreover, in the past few years, the market of Genome Editing has recorded a significant development and is anticipated to further rise.

Market research report for every industry is based on various important factors, for example demand & supply, market trends, revenue growth patterns and market shares. Report on the Global Genome Editing Market is made after comprehensive research conducted by a systematized methodology. These techniques are helpful for analyzing the market in terms of research guidelines. Basically, research reports cover all the information about the consumers, vendors, manufactures, research papers, products and many more. They provide a range of marketing as well as business research solutions basically designed for readers looking forward to invest in the market. Moreover, their research reports are collection of a particular industry research that includes information on products, market size, countries, trends, business research details & much more.

Request Free Sample of This Report at: https://www.thebrainyinsights.com/enquiry/sample-request/12995

The global genome editing market was valued at USD 8.18 billion in 2023, increasing at a CAGR of 17% from 2024 to 2033 and anticipated to reach USD 39.33 billion by 2033.

Some of the major companies that are covered in this report:

Merck KGaA, Thermo Fisher Scientific, Lonza, Sangamo Therapeutics, GenScript, Editas Medicine, Tecan Life Sciences, CRISPR Therapeutics AG, Cellectis S.A, Agilent Technologies, Precision Biosciences, Creative Biogene, Bluebird Bio, PerkinElmer, Regeneron Pharmaceuticals, Intellia Therapeutics, Vigene Biosciences, Transposagen Biopharmaceuticals, Synthego, Beam Therapeutics, Caribou Biosciences, Integrated DNA Technologies, Recombinetics, OriGene Technologies, New England Biolabs

Furthermore, research report covers all the quantitative as well as qualitative aspects about the Genome Editing markets across the globe. The report is also inclusive of different market segmentation, business models and market forecasts. This market analysis enables the manufacturers with impending market trends. A thorough scrutiny of prominent market players or industrialists is vital aspect for planning a business in the market. Also, senableout the rivals enables in attaining valuable data about the strategies, company’s models for business, revenue growth as well as statistics for the individuals attracted towards the market. This report is very useful for the new entrants as it offers them the idea about the different approaches towards the market.

The key factor important for making any new business effective is advancement or making impactful modifications in the business. Report on global keyword market is an extensive paper that covers all the aspects of the market analysis and enables a comprehensive summary to its readers. In a nutshell, the Genome Editing market research reports is a one-stop solution for all requirements by the in-house experts.

Market Segmentation

By Type

by Application:

Animal Genetic Engineering

Cell Line Engineering

Plant Genetic Engineering

by Technology:

TALENs

Zinc Finger Nucleases (ZFNs)

CRISPR/Cas9

By Applications

The Genome Editing Market Report Addresses:

Estimated size of the market

The segment that accounted for a large market share in the past

The segment that is anticipated to account for a dominant market share by 2032?

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The Report Provides:

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Competitive analysis, with company overview, products, revenue, and strategies.

Impartial assessment of the market

Strategic recommendations to help companies increase their market presence

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About The Brainy Insights:

The Brainy Insights is a market research company, aimed at providing actionable insights through data analytics to companies to improve their business acumen. We have a robust forecasting and estimation model to meet the clients' objectives of high-quality output within a short span of time. We provide both customized (clients' specific) and syndicate reports. Our repository of syndicate reports is diverse across all the categories and sub-categories across domains. Our customized solutions are tailored to meet the clients' requirement whether they are looking to expand or planning to launch a new product in the global market.

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[autodidacticpolymath-blog]

A Policy arm @ Cambridge had VERY interesting use of "confidential" patient data for Genomic & Medical Research during and post COVID.

20 15 Pathogen genomics into practice Data sharing to support UK clinical genetics and genomics services Genetic screening programmes: an international review of assessment criteria 2017 Personalised prevention in breast cancer – the policy landscape Developing effective ctDNA testing services for lung cancer Linking and sharing routine health data for research Variant classification and…

[airises]

What is Biotech? Unlocking the Power of Biology

“Unlocking the Power of Biology: Biotech Innovations Transforming Our World” Biotechnology: Revolutionizing Industries and Improving Lives Biotech, short for biotechnology, is a rapidly evolving field that combines biology, genetics, and engineering to develop innovative solutions for various industries and aspects of our lives. From healthcare and agriculture to environment and energy, biotech…

[cailynnjohnson]

genome editing talen

As effective molecular scissors, TALENs are widely used to induce a variety of specific and efficient genomic modifications in various cell types and different model organisms. With technology development, TALs can be engineered to deliver virtually any enzyme to any site and perform gene-editing functions, including gene knockout, knockin, mutagenesis, gene tagging, and for disease and cell therapeutics models. It is documented that TALENs have been used to engineer stably modified human embryonic stem cell and induced pluripotent stem cell (IPSCs), moreover, they have great promises in the cell therapy of cancers and genetic disorders, such as sickle cell disease and xeroderma pigmentosum. Learn more about genome editing talen.