#genome editing

[bitch-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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[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.

[august--and-everything-after]

As far as I'm concerned, the colossal biosciences people are grifters.

Yeah the science they're doing is cool but the way they're presenting it is so overblown and exaggerated that they're practically just lying.

[k20spock]

if i have to see one more article going omg dire wolves have been brought back from extinction!! im gonna strangle somebody

[hyeriyearner]

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

[ivytherium]

To anyone who follows me, I don't care about nor trust Colossal Biosciences anymore (The people behind the "Wooly Mice"). They have proven themselves to be headline-chasing grifters after this latest stunt. They are claiming to have de-extincted *Aenocyon dirus*, aka the Dire Wolf, by editing just 20 genes from the the DNA of a Grey Wolf (*Canis lupus*) to make this thing:

If it wasn't clear from their scientific names, Grey Wolves and Dire Wolves aren't remotely related to one another aside from being Canids, despite what pop culture like Game of Thrones would have you believe. If they did look like each other, it would have had to be via convergent evolution, as they only shared a common ancestor over 5 million years ago.

This distinction, however, isn't found in the publicized articles about this so-called resurrected Dire Wolf and makes their claim that they brought the Dire Wolf back by simply editing *20* genes from the genome of a Grey Wolf laughable. A Dire Wolf would have shared more in common genetically with a Maned Wolf (*Chrysocyon brachyurus*) or Bush Dog (*Speothos venaticus*) than it would with a Grey Wolf.

Bottom line, don't fall for whatever this company is trying to tell you. If the Dire Wolf were to be brought back, it wouldn't be via something like this, and certainly wouldn't *look* like this. If you want an idea as to how a real Dire Wolf would look like in life, here is some fantastic paleoart by artist Mauricio Antón:

Addendum: I seem to have partially miscalculated Dire Wolf genetics. They were not closer to Maned Wolves or Bush Dogs, but they were still not closely related to Grey Wolves. They were basal members of Canini, related to canids like Jackals (genus Lupulella) but distinct from them. I am sorry for this misinformation in my attempt to correct other misinformation. My main point, however, is still correct.

[mrnnki]

I need to draw the space cats doing horizontal gene transfer

[kamalkafir-blog]

ultra-powerful CRISPR treatment trialled in a person

A genome-editing system more versatile than the classic CRISPR–Cas-9 complex (artist’s illustration) has been trialled in an 18-year-old with a genetic immune disorder.Credit: Laguna Design/Science Photo Library The CRISPR family’s most versatile member has made its medical debut: a cutting-edge gene-editing technique known as prime editing has been used to treat a person for the first time. The…

[healthcare-updates-with-sns]

Genome Editing Market: Market Trends and Market Analysis 2024-2032

The Genome Editing Market was valued at USD 6.46 Billion in 2023 and is projected to reach USD 25.99 Billion by 2031, growing at a compound annual growth rate (CAGR) of 18.9% during the forecast period from 2024 to 2031. This remarkable growth is attributed to the increasing demand for precise and effective gene therapies, rapid advancements in CRISPR technology, and a surge in research funding related to genomics and biotechnology.

Market Overview

Genome editing, also known as gene editing, is a powerful tool that allows scientists to alter DNA within organisms. This technique is being widely adopted across healthcare, agriculture, and research sectors to treat genetic disorders, enhance crop traits, and study gene functions. The growth of the genome editing market is being accelerated by rising investments in R&D, increasing prevalence of genetic diseases, and expanding applications in drug development and regenerative medicine.

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Regional Analysis

North America currently dominates the genome editing market due to its well-established healthcare infrastructure, strong presence of key industry players, and robust investment in genetic research. The region's leadership in adopting advanced technologies and favorable regulatory policies supports ongoing innovation.

Europe follows closely, benefiting from substantial government initiatives and funding for genomic research. Meanwhile, the Asia-Pacific region is emerging as a lucrative market, driven by increasing healthcare expenditure, a growing biotechnology sector, and supportive government policies in countries like China, Japan, and India.

Market Segmentation

The genome editing market is segmented based on:

Technology: CRISPR, TALEN, ZFN, Antisense, and Others

Application: Cell Line Engineering, Animal Genetic Engineering, Plant Genetic Engineering, and Others

End-Use: Pharmaceutical & Biotechnology Companies, Academic & Research Institutes, and Contract Research Organizations (CROs)

CRISPR continues to lead the technology segment due to its simplicity, efficiency, and cost-effectiveness compared to other genome editing tools.

Key Players

Merck KGaA - CRISPR-Cas9 Gene Editing Platform

Cibus Inc. - Rapid Trait Development System (RTDS)

Recombinetics - Gene Editing for Animal Health and Agricultural Biotechnology

Sangamo Therapeutics - Zinc Finger Nucleases (ZFNs)

Editas Medicine - EDIT-101 (CRISPR-based Gene Therapy)

Precision BioSciences - Arcus Gene Editing Platform

CRISPR Therapeutics - CTX001 (CRISPR-based Therapy for Genetic Disorders)

Intellia Therapeutics, Inc. - NTLA-2001 (CRISPR-based Therapeutic)

Caribou Biosciences, Inc. - CRISPR-Cas12a and CRISPR-Cas9 Technologies

Cellectis S.A. - TALEN Gene Editing System

AstraZeneca - CRISPR Gene Editing Research Initiatives

Takara Bio Inc. - Gibson Assembly and CRISPR/Cas9 Systems

Horizon Discovery Ltd. (Revvity, Inc.) - GENESIS Gene Editing Services

Danaher Corporation - EditR (Gene Editing Platform)

Transposagen Biopharmaceuticals, Inc. - Transposon-based Gene Editing Technologies

Genscript Biotech Corp - CRISPR Gene Editing Kits and Services

New England Biolabs - CRISPR Enzymes and Tools

OriGene Technologies, Inc. - CRISPR/Cas9 Gene Editing Tools

Bluebird Bio, Inc. - LentiGlobin (Gene Therapy for Genetic Disorders)

Lonza - Lonza’s Genome Editing Services

Thermo Fisher Scientific, Inc. - GeneArt CRISPR Nuclease Kits and Services

Key Points:

Market projected to grow at a CAGR of 18.9% from 2024 to 2031

CRISPR remains the dominant technology with broad research and therapeutic use

Rising prevalence of genetic disorders fueling demand for genome editing tools

North America holds the largest market share, followed by Europe and Asia-Pacific

Increased public and private funding supports R&D in genome editing

Expanding application across agriculture, medicine, and biotech research

Future Scope

The future of the genome editing market looks promising, with significant growth potential in personalized medicine, gene therapy, and synthetic biology. Innovations in delivery systems, ethical regulation development, and expanding clinical applications are expected to shape the next phase of market evolution. Additionally, collaborations between biotech firms, academic institutions, and regulatory bodies will accelerate the transition of genome editing from the lab to mainstream healthcare.

Conclusion

With its transformative impact on healthcare and agriculture, the genome editing market is poised for substantial growth over the coming years. Technological innovations, supportive regulatory frameworks, and increasing global investment in genomics are set to propel the industry into a new era of precision science and personalized treatment.

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