#Crispr technology

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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.

[rightnewshindi]

HIV News; एम्स्टर्डम यूनिवर्सिटी मेडिकल सेंटर के विशेषज्ञों का बड़ा दावा, क्रिस्प तकनीक से खत्म किया एचआईवी वायरस

HIV News; एम्स्टर्डम यूनिवर्सिटी मेडिकल सेंटर के विशेषज्ञों का बड़ा दावा, क्रिस्प तकनीक से खत्म किया एचआईवी वायरस

HIV Breakthrough: एम्स्टर्डम यूनिवर्सिटी मेडिकल सेंटर से जुड़े विशेषज्ञों ने दावा ��िया है कि उन्होंने लैब प्रयोगों के दौरान संक्रमित कोशिकाओं से एचआईवी को सफलतापूर्वक खत्म कर दिया है। उन्होंने जिस प्रक्रिया का उपयोग किया वह शब्द क्रिस्प है। क्रिस्प डीएनए स्ट्रैंड को काटने के लिए विशेष एंजाइमों का उपयोग करता है, यह उसी तरह काम करता है जिस तरह कैंची काम करती है। क्रिस्प से किया गया वायरस…

[americanmysticom]

LET’S LEARN ABOUT CRISPR TECHNOLOGY

The daring[?] Chinese biophysicist who created the world’s first gene-edited children has been set free after three years in a Chinese prison.

He Jiankui created shock waves in 2018 with the stunning claim that he’d altered the genetic makeup of IVF embryos and implanted them into a woman’s uterus, leading to the birth of twin girls. A third child was born the following year.

Following international condemnation of the experiment, He was placed under home arrest and then detained. In December 2019, he was convicted by a Chinese court, which said the researcher had “deliberately violated” medical regulations and had “rashly applied gene editing technology to human assisted reproductive medicine.”

https://www.technologyreview.com/2022/04/04/1048829/he-jiankui-prison-free-crispr-babies/

See Also; Gene Editing + The CRISPR Curse childrenshealthdefense Published November 10, 2022 https://rumble.com/v1tag4a-gene-editing-the-crispr-curse.html

700 Million Worldwide Will Die from CV19 Vax by 2028 – Dr. David Martin Greg Hunter's USAWatchdog.com Published June 28, 2022 https://rumble.com/v1acoaa-up-to-100-million-will-die-from-cv19-vax-by-2028-dr-david-martin.html

download mp4; https://icedrive.net/s/XvAxN8ht469Bfai7kyAfQXbC8xjb

https://prosecutenow.io/

see also: https://covid19criticalcare.com/treatment-protocols/

[macleod]

New CRISPR cancer treatment tested in humans for first time

Researchers have made an important step forward toward a long-desired goal: using the gene-editing technology CRISPR to treat cancer. [Source: Time]

Now, PACT Pharma and UCLA have used CRISPR to remove and add genes to these cells to help them recognize a patient’s specific tumor cells. [...]

By genetically engineering T cells to be better at spotting proteins commonly found on the surfaces of blood cancer cells, researchers have been able to develop treatments — called “CAR-T cell therapies” — for people with those cancers. [..]

Using CRISPR, they knocked out a gene for an existing receptor and inserted a gene for a cancer-targeting receptor into T cells that lacked it. Once they had engineered what they thought would be enough T cells, the researchers infused them back into the patient.

The results: Later biopsies found that up to 20% of the immune cells in the patients’ tumors were the engineered T cells, suggesting that those cells were in fact very adept at homing in on the cancer.

Only two of the 16 participants experienced minor side effects — fever, chills, confusion — attributable to the T cells, but they quickly resolved. [..]

A month after treatment, five of the patients’ tumors were the same size as before, suggesting that the engineered cells may have had a stabilizing effect on their condition.

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My thoughts: While the results weren't absolutely mind-staggering, 100% fantastic, the amount that they were able to keep the cancer cells at bay, and normalize the growth is tremendously great.

The researchers went on to say that this may be due to their purposeful limited attack methods, and that this is something they can build on and truly develop a personalized life-saving or extending treatment for cancer.

“We are reprogramming a patient’s immune system to target their own cancer, [..] It’s a living drug, so you can give one dose and ideally have life-long protection [from the cancer].”

Source: Freethink, November 21st, 2022.

[globalcat]

From Jellyfish to Glowing Cats: The Journey of Bioluminescence in Pets and the ethical questions

Glowing Cats for science. Is it the future also for pets?

Glowing pets have captivated both the scientific community and the public’s imagination, presenting a fascinating intersection between biotechnology and domestic animals. This phenomenon can be observed in two distinct categories: natural bioluminescence and genetically modified organisms (GMOs). As we delve into this topic, we’ll explore the science behind glowing pets, the ethical…

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License plates of MIT

New Post has been published on https://thedigitalinsider.com/license-plates-of-mit/

License plates of MIT

What does your license plate say about you?

In the United States, more than 9 million vehicles carry personalized “vanity” license plates, in which preferred words, digits, or phrases replace an otherwise random assignment of letters and numbers to identify a vehicle. While each state and the District of Columbia maintains its own rules about appropriate selections, creativity reigns when choosing a unique vanity plate. What’s more, the stories behind them can be just as fascinating as the people who use them.

It might not come as a surprise to learn that quite a few MIT community members have participated in such vehicular whimsy. Read on to meet some of them and learn about the nerdy, artsy, techy, and MIT-related plates that color their rides.

A little piece of tech heaven

One of the most recognized vehicles around campus is Samuel Klein’s 1998 Honda Civic. More than just the holder of a vanity plate, it’s an art car — a vehicle that’s been custom-designed as a way to express an artistic idea or theme. Klein’s Civic is covered with hundreds of 5.5-inch floppy disks in various colors, and it sports disks, computer keys, and other techy paraphernalia on the interior. With its double-entendre vanity plate, “DSKDRV” (“disk drive”), the art car initially came into being on the West Coast.

Sam Klein works on replacing the floppy disks on his art car, which can often be seen around the MIT campus sporting a “DSKDRV” license plate.

Photo: Courtesy of Sam Klein

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Klein, a longtime affiliate of the MIT Media Lab, MIT Press, and MIT Libraries, first heard about the car from fellow Wikimedian and current MIT librarian Phoebe Ayers. An artistic friend of Ayers’, Lara Wiegand, had designed and decorated the car in Seattle but wanted to find a new owner. Klein was intrigued and decided to fly west to check the Civic out.

“I went out there, spent a whole afternoon seeing how she maintained the car and talking about engineering and mechanisms and the logistics of what’s good and bad,” Klein says. “It had already gone through many iterations.”

Klein quickly decided he was up to the task of becoming the new owner. As he drove the car home across the country, it “got a wide range of really cool responses across different parts of the U.S.”

Back in Massachusetts, Klein made a few adjustments: “We painted the hubcaps, we added racing stripes, we added a new generation of laser-etched glass circuits and, you know, I had my own collection of antiquated technology disks that seemed to fit.”

The vanity plate also required a makeover. In Washington state it was “DISKDRV,” but, Klein says, “we had to shave the license plate a bit because there are fewer letters in Massachusetts.”

Today, the car has about 250,000 miles and an Instagram account. “The biggest challenge is just the disks have to be resurfaced, like a lizard, every few years,” says Klein, whose partner, an MIT research scientist, often parks it around campus. “There’s a small collection of love letters for the car. People leave the car notes. It’s very sweet.”

Marking his place in STEM history

Omar Abudayyeh ’12, PhD ’18, a recent McGovern Fellow at the McGovern Institute for Brain Research at MIT who is now an assistant professor at Harvard Medical School, shares an equally riveting story about his vanity plate, “CRISPR,” which adorns his sport utility vehicle.

The plate refers to the genome-editing technique that has revolutionized biological and medical research by enabling rapid changes to genetic material. As an MIT graduate student in the lab of Professor Feng Zhang, a pioneering contributor to CRISPR technologies, Abudayyeh was highly involved in early CRISPR development for DNA and RNA editing. In fact, he and Jonathan Gootenberg ’13, another recent McGovern Fellow and assistant professor at Harvard Medical School who works closely with Abudayyeh, discovered many novel CRISPR enzymes, such as Cas12 and Cas13, and applied these technologies for both gene therapy and CRISPR diagnostics.

So how did Abudayyeh score his vanity plate? It was all due to his attendance at a genome-editing conference in 2022, where another early-stage CRISPR researcher, Samuel Sternberg, showed up in a car with New York “CRISPR” plates. “It became quite a source of discussion at the conference, and at one of the breaks, Sam and his labmates egged us on to get the Massachusetts license plate,” Abudayyeh explains. “I insisted that it must be taken, but I applied anyway, paying the 70 dollars and then receiving a message that I would get a letter eight to 12 weeks later about whether the plate was available or not. I then returned to Boston and forgot about it until a couple months later when, to my surprise, the plate arrived in the mail.”

While Abudayyeh continues his affiliation with the McGovern Institute, he and Gootenberg recently set up a lab at Harvard Medical School as new faculty members. “We have continued to discover new enzymes, such as Cas7-11, that enable new frontiers, such as programmable proteases for RNA sensing and novel therapeutics, and we’ve applied CRISPR technologies for new efforts in gene editing and aging research,” Abudayyeh notes.

As for his license plate, he says, “I’ve seen instances of people posting about it on Twitter or asking about it in Slack channels. A number of times, people have stopped me to say they read the Walter Isaacson book on CRISPR, asking how I was related to it. I would then explain my story — and describe how I’m actually in the book, in the chapters on CRISPR diagnostics.”

Displaying MIT roots, nerd pride

For some, a connection to MIT is all the reason they need to register a vanity plate — or three. Jeffrey Chambers SM ’06, PhD ’14, a graduate of the Department of Aeronautics and Astronautics, shares that he drives with a Virginia license plate touting his “PHD MIT.” Professor of biology Anthony Sinskey ScD ’67 owns several vehicles sporting vanity plates that honor Course 20, which is today the Department of Biological Engineering but has previously been known by Food Technology, Nutrition and Food Science, and Applied Biological Sciences. Sinskey says he has both “MIT 20” and “MIT XX” plates in Massachusetts and New Hampshire.

Top: Stellar license plates of professor of the practice emerita Marcia Bartusiak and her husband, Stephen Lowe PhD ’88. Bottom: Anthony Sinskey ScD ’67 honors MIT Course 20 on two of his vehicles.

Photo: MIT News

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At least two MIT couples have had dual vanity plates. Says Laura Kiessling ’83, professor of chemistry: “My plate is ‘SLEX.’ This is the abbreviation for a carbohydrate called sialyl Lewis X. It has many roles, including a role in fertilization (sperm-egg binding). It tends to elicit many different reactions from people asking me what it means. Unless they are scientists, I say that my husband [Ron Raines ’80, professor of biology] gave it to me as an inside joke. My husband’s license plate is ‘PROTEIN.’”

Professor of the practice emerita Marcia Bartusiak of MIT Comparative Media Studies/Writing and her husband, Stephen Lowe PhD ’88, previously shared a pair of related license plates. When the couple lived in Virginia, Lowe working as a mathematician on the structure of spiral galaxies and Bartusiak a young science writer focused on astronomy, they had “SPIRAL” and “GALAXY” plates. Now retired in Massachusetts, while they no longer have registered vanity plates, they’ve named their current vehicles “Redshift” and “Blueshift.”

Still other community members have plates that make a nod to their hobbies — such as Department of Earth, Atmospheric and Planetary Sciences and AeroAstro Professor Sara Seager’s “ICANOE” — or else playfully connect with fellow drivers. Julianna Mullen, communications director in the Plasma Science and Fusion Center, says of her “OMGWHY” plate: “It’s just an existential reminder of the importance of scientific inquiry, especially in traffic when someone cuts you off so they can get exactly two car lengths ahead. Oh my God, why did they do it?”

Are you an MIT affiliate with a unique vanity plate? We’d love to see it!

[techtoio]

The Latest Breakthroughs in Technology for 2024

Introduction

Welcome to TechtoIO, your go-to source for the latest in tech news and trends. As we dive into 2024, the tech landscape continues to evolve at a rapid pace. From artificial intelligence advancements to breakthroughs in quantum computing, this year promises to be a landmark in technological innovation. In this comprehensive guide, we’ll explore the most exciting developments in technology for 2024, offering insights into how these advancements will impact our lives and industries. Read to continue

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AI Decoded

Two days ago, I commented on an interview @dylan_curious that was about AI and how it could save us.?? My comment included CRISPR technology, and this morning I found this video that shows how CRISPR is being used to manipulate the human genome. They say that CRISPR technology is being used to rid humans of genetic disorders. My question is how long until it is made public that they are using…

[airises]

How Biotech is Revolutionizing Stem Cell Research: Insights and Innovations

fields that have become increasingly intertwined. As we delve deeper into the potential of stem cells, biotechnology companies are at the forefront, developing innovative technologies and techniques that could potentially revolutionize treatments for various diseases and conditions. This article provides a comprehensive overview of how biotechnology is influencing stem cell research, detailing…

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https://cynochat.com/read-blog/181726_crispr-technology-market-size-analysis-and-forecast-2031.html

[ndegegeoffrey]

CRISPR; Are We Pushing The Gene Technology Too Far?

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DAILY DOSE: NASA's Mars helicopter permanently grounded; Study estimates 64,000 pregnancies by rape in states with abortion bans.

INGENUITY’S RUN COMES TO AN END. NASA’s Ingenuity Mars Helicopter has concluded its groundbreaking mission on Mars after achieving far beyond its initial objectives. Originally slated for five test flights over 30 days, Ingenuity impressively completed 72 flights in almost three years, exceeding its planned distance fourteen-fold and accumulating over two hours of flight time. Despite a damaged…

[techtoio]

Biotech Breakthroughs: Cutting-Edge Innovations That Will Change Health Care

Introduction

The field of biotechnology is at the forefront of some of the most groundbreaking advancements in healthcare. With new discoveries and innovations emerging at an unprecedented rate, biotech is poised to transform the way we approach health care. From revolutionary treatments to personalized medicine, the potential of biotech to improve lives is immense. In this article, we’ll delve into the most significant biotech breakthroughs and how they are set to change health care as we know it. Read to continue