#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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[nonbinary-potatoes]

sorry to put this on ur dash

you should help me with my research paper...

"why?" you may be asking...

plsplsplsplsplsplsplsplspls 🥺

this is the link, any answers are greatly appreciated

[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…

[invinciblevalentine]

hojo pisses me tf off. Like yeah yeah human rights violations unethical science blah blah blah HAVE WE CONSIDERED THE DAMAGE HE’S BEEN DOING TO THE FF7 SCIENTIFIC COMMUNITY????

Listen he’d be a morally grey character if his work led to reproducible results that could benefit the rest of humanity but GOOD GAIA does he not even do that.

his sample sizes are SHIT. his experimental design is SHIT. his documentation is SHIT. his biases are VISIBLE. I’m astral projecting into the ff7 universe just to strangle this man and take away whatever diploma he got bc clearly he hired someone to do his PhD for him.

AND HE INSPIRED SO MANY TO FOLLOW IN HIS SHITTY SHITTY FOOTSTEPS. like the only two things we could even marginally call reproducible is his work in making the SOLDIER program and Fuhitos attempt to replicate it with his RAVENs. and even then there were no improvements on the procedure or attempts to create a procedure that left the patients in better health.

which is a CRIMINALLY STUPID THING TO DO TO YOUR GIANT SUPER SOLDIER ARMY. WYM THAT INSANITY IS JUST A POSSIBLE SIDE EFFECT?????

an argument could be made that we just don’t SEE hojos documentation and shit bc we’re playing through the eyes of people who don’t know all that shit.

HOWEVER. if hojo was actually doing any of that he’d have spotted the degradation problem in his animal models first.

BECAUSE WHO THE FUCK JUMPS YO HUMAN SUBJECTS??? ITS NOT JUST UNETHICAL ITS EXTREMWLY EXPENSIVE TO MAINTAIN THE WELLBEING OF AND ACQUIRE A WHOLE HUMAN PERSON.

if he’d started with, like, mouse models to demonstrate how mako treatments affect mammals, he’d have gotten so much more work done and achieved more reliable results. WITH A FRACTION OF THE COST.

BUT NOOOOOOOOOO. APPARENTLY SHINRA SHITS MONEY SO WHO CARES ABOUT SAMPLE SIZES AND STATISTICAL TESTS AND REPRODUCIBLE RESULTS!!

HOJO IS A HACK AND A FRAUD AND A SHIT SCIENTIST TO BOOT. WATCH YOUR BACK BITCH YOUVE HOED YOUR LAST JO.

[flooding-your-timeline]

hi okay so this might be a hot take. ai sucks ass with respect to pretty much every use it has right now - EXCEPT medicine.

it's being used on a high scale to test out different variables for so many new treatments, vaccines, meds, where you can get a hundred thousand possible simulations of a situation within a. idk. day?

obviously it's not being relied on completely. there's so many procedures to be followed, for safety, but it's speeding things up, there - in one of the most important fields.

[sybaritick]

jokes about omii and I keeping someone as a pet crossed over into not passing as ironic any more years ago now but who among us has not posted several multi-paragraph text posts about catboy ownership

[whyamiherewhosummonedme]

I am proud to serve.

what is THE worst thing you've ever drank. all liquids acceptable. please tell me what it was, bonus points for why

[healthcaremmr]

The Genome Editing Market size was valued at USD 7.23 Bn in 2023 and market revenue is growing at a CAGR of 15.8 % from 2023 to 2030, reaching nearly USD 20.19 Bn by 2030.

[farmerstrend]

Genome Editing in Agriculture: How Scientists Are Creating Disease-Resistant Rice in East Africa

The “Healthy Crops” consortium, an international team of researchers, which includes Heinrich Heine University Düsseldorf (HHU), has – in collaboration with the Kenya Agricultural and Livestock Research Organization (KALRO) – developed an innovative strategy to combat the disease Bacterial Blight (for short: BB) in rice using genome editing technology. If approved for use by farmers in Kenya, the…

[otto-von-stirlitz]

#they are never ever going to successfully gene edit elephants to look like mammoths and i'm sure everyone on this project knows that#for one we don't have anything close to a completely mapped genome for elephants the way we do for mice#that's why we use mice for gene experiments! also they require very few resources to house and you can use thousands of them at once#and they have short pregnancies and many offspring and a high tolerance for inbreeding. you'll notice none of this is true for elephants#this is 100% a promotional stunt. they'll get more funding for whatever they're *really* trying to do. maybe genome mapping?#all they have done is slightly alter the fur quality of a furred animal into a variant that already exists (angora)#but yes they are very cute! (x)

SO THEY MADE WOOLY MICE USING MAMMOTH GENOMES AND I LOVE THEM