Latest news with #CRISPR-Cas9
New York Post
2 days ago
- Health
- New York Post
Could Down syndrome be eliminated? Scientists say cutting-edge gene editing tool could cut out extra chromosome
Cutting-edge gene editing technology could eradicate Down syndrome, according to Japanese scientists. Down syndrome, which causes a range of developmental differences and affects 1 in 700 newborns in the United States, is caused by the presence of an extra copy of chromosome 21. The extra chromosome, also known as trisomy 21, causes cellular overactivity, compromises a range of processes within the body, and can manifest in distinctive physical traits, learning difficulties, and health concerns. Advertisement Now new research out of Mie University in Japan suggests that by using the DNA-modifying tech CRISPR, it is possible to remove the surplus chromosome in affected cells and bring cellular behavior closer to typical function. 3 Down syndrome, which causes a range of developmental differences and affects 1 in 700 newborns in the United States, is caused by the presence of an extra copy of chromosome 21. Mongkolchon – CRISPR-Cas9 is a gene-editing system that utilizes an enzyme to identify specific DNA sequences. Once the enzyme locates a matching site, it snips through the DNA strands. Ryotaro Hashizume and his colleagues designed CRISPR guides to target only the trisomy 21 chromosome, a process called allele-specific editing, which directs the cutting enzyme to the desired spot. Advertisement When they used it on lab-grown cells, removing the extra copy of the gene normalized the way the genes expressed themselves in the body — suggesting that the genetic burden had been removed. They also found that after the extra chromosome was removed, genes tied to nervous system development were more active and those related to metabolism were less active. This backs up previous research that found extra copies of chromosome 21 disrupt brain development during early fetal growth. Researchers also tested their CRISPR guides on skin fibroblasts, which are mature, non-stem cells taken from people with Down syndrome. Advertisement In these fully developed cells, the editing method successfully removed the extra chromosome in a number of cases. 3 Hashizume and his team designed CRISPR guides to target only the trisomy 21 chromosome, a process called allele-specific editing, which directs the cutting enzyme to the desired spot. Gorodenkoff – After removal, these corrected cells grew faster and had a shorter doubling time than untreated cells, suggesting that removing the extra chromosome may help with the biological strain that slows down cell growth. Advertisement But the CRISPR can affect healthy chromosomes, too, and researchers are refining their program so that it only attaches to the extra copy of chromosome 21. This work proves that, rather than making small fixes, CRISPR can eliminate an entire chromosome. The scientists published their findings in PNAS Nexus. Hashizume and his team are hopeful that their work may be used to design regenerative therapies and treatments that address genetic surplus at its source. 3 Researchers also tested their CRISPR guides on skin fibroblasts, mature, non-stem cells taken from people with Down syndrome. Yakobchuk Olena – Researchers will continue to analyze the risks of DNA changes and monitor how modified cells function over time and their viability in real-world settings. A recent case study explored a medical mystery related to Down syndrome; the brain of an American woman with Down syndrome showed all the classic signs of Alzheimer's disease, yet she remained symptom-free throughout her lifetime. Advertisement People with Down syndrome face a much higher risk of developing Alzheimer's-related dementia as they age — an estimated three to five times higher than the general population. Scientists are still working to pinpoint the exact cause, but it's believed that the extra copy of chromosome 21 drives the overproduction of amyloid precursor protein. This excess production leads to the buildup of amyloid beta plaques in the brain, a hallmark of Alzheimer's disease.
Time of India
12-06-2025
- Health
- Time of India
AI driven Innovations in targeted drug delivery: The emergence of needless syringes.
How do they work? Live Events Developed using AI, more specifically DeepMind's AlphaFold program, there now exists a syringe that does not require a long stainless steel needle that should pierce through skin and administer drugs into the targeted area. Although this innovation remains in its infancy, the tests conducted in vitro and in mice have shown highly promising so, this should revolutionize targeted drug delivery and gene therapies, potentially reshaping the biotechnology and pharmaceutical sectors. Engineering needless syringes (molecular syringes) for direct protein injection into human cells shall enhance precision and minimize adverse effects. Moreover, this innovation addresses a major bottleneck in medicine, effectively delivering therapeutic molecules to the right novel approach uses a naturally occurring syringe-like protein from "photorhabdus' bacteria, with which AI helps to target specific cell receptors. These bacteria utilize syringe-like machines, called 'extracellular contractile injection systems (eCISs),' to inject proteins into host cells. Researchers have modified these eCISs to be approximately 100 nanometers long to deliver the proteins into human process further inculcates re-engineering the tail fibers of the eCIS to latch to a specific receptor on the surface of the target cells, allowing the syringe to cling onto these cells. AlphaFold, here, is used to enhance the binding affinity and increase the precision with which the tail fibers will latch themselves. Once attached, the syringe shall inject its protein material through the cell membrane. Scientists can manipulate another part of the complex to control the delivery of specific proteins with high can also be engineered to recognize and bind to specific proteins on mouse cells (in a mouse model), ensuring that the injected material is delivered only to targeted areas, all while avoiding unwanted immune responses. This breakthrough holds immense potential for revolutionizing gene therapy, cancer treatment, and biocontrol by enabling highly precise and controlled drug the AI-designed molecular syringes were also able to demonstrate their capability to deliver a variety of therapeutic agents, like the CRISPR-Cas9, a gene-editing tool often described as a molecular "scissor" that facilitates precise gene editing by cutting specific DNA segments. Moreover, it was able to inculcate the 'zinc-finger deaminases' that enabled 'base-editing' by converting specific DNA bases without cutting the DNA strand. Lastly, it was able to target and kill cancer cells by delivering cytotoxic agents directly into integration of DeepMind's AlphaFold was significant in redesigning the tail fibers of the molecular syringe. This tool allowed researchers to predict protein structures at a much faster rate, accelerating the process of modifying the syringe to target human cells. The study's first author, Joseph Kreitz, emphasized that without AlphaFold, achieving such predicted structures would have taken much longer, extending up to many more months, whereas the AI allowed for near real-time predictions and therefore streamlined the engineering from their current applications, researchers see these molecular syringes as programmable delivery devices. Devices of this kind could allow for controlled dosing, sustained release of therapeutics, and enhanced treatments like chemotherapy by reducing toxicity and increasing efficiency. Therefore, fostering a potential for these syringes to combat pathogenic bacteria by delivering antibacterial agents directly into harmful microbes.
Indian Express
05-06-2025
- Science
- Indian Express
Meet India's first gene-edited sheep, born in a Srinagar lab, now a healthy six-month-old
Six months ago, Professor Riyaz Ahmad Shah and his team at the Embryo Biotechnology Lab of Sher-e-Kashmir University of Agricultural Sciences and Technology (SKUAST) in Srinagar celebrated a quiet success – the birth of a Kashmir Merino, India's first gene-edited sheep. But the team didn't rush to announce its arrival to the world – they waited, given the inherent uncertainties of scientific breakthroughs. The announcement was finally made last week after the results were validated by gene sequencing and standardised. 'This marks a new era in genetic research and has put us on the future path of transgenics in animals (inserting a foreign gene in an animal),' says Prof Shah, Dean of the Faculty of Veterinary Sciences and Animal Husbandry at SKUAST. Gene-editing of livestock animals is a growing area of research in India with significant applications targeted at enhanced meat yield and milk production, disease resistance and resilience to impacts of climate change. Prof Shah and his team edited the sheep's myostatin gene. 'This particular gene is a negative growth regulator. By targeting this gene, we can increase the muscle mass of a sheep by 30 per cent,' says Prof Shah. Talking about the significance of the project, Dr Naresh Selokar, Senior Scientist, Animal Biotechnology at National Dairy Research Institute (NDRI) in Karnal, says, 'In the Indian context, this (gene-editing of sheep) is a very significant achievement, especially considering the trait (gene) that has been targeted. 'Given our population and the huge demand for meat, without gene-editing, it is impossible to change the trait of a farm animal or to make them disease resistant. This is already an easy and approved method for production globally… In India, we need to have more high-quality, disease-resistant produce through gene editing,' says Dr Selokar, who is credited with developing the first gene-edited embryo of a buffalo in 2024. It was in 2020 that Prof Shah and his core team at SKUAST's Embryo Biotechnology Lab — Dr Suhail Magray, Dr Muneer Dar, Dr Younus Farooq, Dr Nida Handoo, Dr Syed Hilal, Dr Abrar and Dr Nafis — embarked on their ambitious project. The embryo of the sheep was first kept under laboratory conditions for some time and then transferred to a surrogate mother, before being gene-edited in July last year. To edit the myostatin gene of the sheep, the team used CRISPR-Cas9, the genome editing technology, which won Emmanuelle Charpentier and Jennifer Doudna the Nobel Prize in Chemistry that very year. The gene-edited sheep was finally born in December last year. It's now a healthy six-month-old, housed at a farm on the Shuhama campus of the Faculty of Veterinary Science. The researchers say it weighed 3.15 kg at birth and has gained 'significant weight' since then. 'We specifically chose the myostatin gene since the goal is to increase the muscle mass of sheep. Any technological advancement in livestock and agriculture is meant to increase the farmer's income and this is an important step towards that,' says Prof Shah. Their journey, says Dr Suhail Magray, wasn't without hurdles. 'We tried different techniques to get the desired results. We failed the first three times, before we got the breakthrough,' he says. In a span of 15 years, SKUAST's Centre of Animal Biotechnology has taken a leap from cloning to gene-editing. In 2012, when the lab was in its infancy, it developed the world's first pashmina goat clone, which it named Noori, using the somatic cell nuclear transfer technique. The birth of Noori was seen as a breakthrough in cloning of endangered species. Noori was born on March 9, 2012 and died in March 2023 when it was 11 years old — the average age for the pashmina goat. Their lab is now working on another gene edit — FGF5 (Fibroblast growth factor) — that will help improve the fibre quality of sheep. 'The beginning has been made,' says Dr Magray. 'Now, we can work on other genes as well that would help to make the animals disease resistant.' With the success of their latest project, Prof Shah and his team are already preparing for the next leap — transgenics. 'We are already working on combining cloning with gene-editing to enhance the technique but our next step is to move towards transgenics,' says Prof Shah. 'Transgenics is important if we are to produce proteins of therapeutic importance – if we can, for instance, produce protein in the milk of an animal, that animal will work as a factory of proteins; animals can be pharmaceutical factories, we can have anti-cancer drugs. But for that, we have to have controlled conditions and bio-secure zones.' A pioneer in genetics, Prof Shah was a PhD student at NDRI, Karnal, and was part of the team that's credited with the first buffalo cloning in the world. The buffalo, Samrupa, didn't survive and six months later, the team developed the second buffalo clone, 'Garima'. Bashaarat Masood is a Special Correspondent with The Indian Express. He has been covering Jammu and Kashmir, especially the conflict-ridden Kashmir valley, for two decades. Bashaarat joined The Indian Express after completing his Masters in Mass Communication and Journalism from the University in Kashmir. He has been writing on politics, conflict and development. Bashaarat was awarded with the Ramnath Goenka Excellence in Journalism Awards in 2012 for his stories on the Pathribal fake encounter. ... Read More
Yahoo
31-05-2025
- Health
- Yahoo
Scientists Gene-Hack Spider to Produce Bright-Red Silk
Researchers used the popular gene-editing technique CRISPR to modify the DNA sequences of house spiders, causing them to produce red fluorescent silk. Scientists are hoping that the US Navy and Air Force-funded research could lead to the development of new "supermaterials" produced by arachnids, Fast Company reports. As detailed in a paper published in the journal Angewandte Chemie, a team of researchers at the University of Bayreuth in Germany injected the eggs of unfertilized female spiders with a CRISPR-Cas9 solution to insert a gene sequence for a red fluorescent protein. After mating with males of the same species, the offspring produced red, fluorescent silk, demonstrating that the experiment had been successful. "Considering the wide range of possible applications, it is surprising that there have been no studies to date using CRISPR-Cas9 in spiders," said senior author and University of Bayreuth professor Thomas Scheibel in a statement. "We have demonstrated, for the first time worldwide, that CRISPR-Cas9 can be used to incorporate a desired sequence into spider silk proteins, thereby enabling the functionalisation of these silk fibres." Apart from turning their silk bright red, the researchers also attempted to knock out a gene called sine oculis, which is responsible for the development of spider eyes. They found that the gene edit caused total or partial eye loss in experiments, highlighting its important role in visual development. By applying CRISPR-Cas9, a technique that has already been widely used to create custom medical treatments or make farm animals more resilient to diseases, the researchers are hoping to come up with a new generation of silk fibers. "Successful spider silk engineering in vivo will, therefore, help to develop and employ new fiber functionalities for a broad range of applications," the team wrote in its paper. "So far, genetic modifications in spiders have been only aimed at evolutionary and developmental research." As Fast Company points out, materials scientists have already been investigating the tactile strength of the silk produced by gene-modified silkworms. But thanks to cutting-edge gene-editing techniques, researchers could soon harness the unique advantages of spider silk as well. While the researchers didn't single out specific use cases for future "supermaterials," the possible applications are practically endless, from lightweight body armor to ultralight running shoes. "The ability to apply CRISPR gene-editing to spider silk is very promising for materials science research — for example, it could be used to further increase the already high tensile strength of spider silk," Scheibel explained. More on CRISPR: In a World First, CRISPR Drug Tailored for One Baby Shows Life-Saving Promise
Indian Express
28-05-2025
- Health
- Indian Express
Daily subject-wise quiz : Science and Technology MCQs on the role of trees near a volcano in an eruption, CRISPR and more (Week 112)
UPSC Essentials brings to you its initiative of subject-wise quizzes. These quizzes are designed to help you revise some of the most important topics from the static part of the syllabus. Attempt today's subject quiz on Science and Technology to check your progress. 🚨 Click Here to read the UPSC Essentials magazine for May 2025. Share your views and suggestions in the comment box or at With reference to the CRISPR (clustered regularly interspaced short palindromic repeats), consider the following statements: 1. It is an immune system found in microbes such as bacteria which fights invading viruses. 2. When a virus infects a bacterial cell, CRISPR helps to establish a memory in the form of antibodies, like in humans. 3. Base editing is a new version of the CRISPR-Cas9 technology. How many of the statements given above are correct? (a) Only one (b) Only two (c) All three (d) None Explanation — A nine-month-old child born with a rare genetic disease has become the first (known) person to successfully get personalised gene-editing treatment, according to a paper published on May 15 in the New England Journal of Medicine. — To treat him, scientists and doctors from the University of Pennsylvania and the Children's Hospital of Philadelphia developed a personalised treatment based on 'base editing', a new version of the decade-old CRISPR-Cas9 technology. Hence, statement 3 is correct. — Scientists believe this method has the potential to treat thousands of unusual genetic illnesses. However, there are significant obstacles to its widespread acceptance. What is CRISPR? — After being infected with a virus, individuals develop an 'immune memory' in the form of antibodies. When they are infected with the same virus again, these antibodies readily recognise and neutralise the pathogens. — CRISPR, an immunological mechanism found in microbes such as bacteria that fights invading viruses, stands for 'clustered regularly interspaced short palindromic repeats'. When a virus infects a bacterial cell, CRISPR contributes to the establishment of a memory — but one that is genetic rather than in the form of antibodies, as in humans. Hence, statement 1 is correct and statement 2 is not correct. — When a virus infects a bacterial cell, the bacterium removes a portion of the virus's genome and inserts it into its own genome. CRISPR then creates a new 'guide' RNA using the freshly acquired DNA. — During a subsequent attack by the same virus, the guide RNA immediately detects and attaches to virus DNA. The guide RNA then instructs an enzyme (a type of protein) called Cas9 to serve as 'molecular scissors' to cut and remove the virus DNA. Therefore, option (b) is the correct answer. With reference to the Tianwen-2 mission, consider the following statements: 1. This mission will be launched by Japan, being its first mission to survey and sample a near-Earth asteroid. 2. It will investigate an asteroid called 469219 Kamo'oalewa, which orbits the Sun at a distance relatively close to Earth. Which of the statements given above is/are correct? (a) 1 only (b) 2 only (c) Both 1 and 2 (d) Neither 1 nor 2 Explanation — China will launch its first mission to survey and sample a near-Earth asteroid this week. Known as the Tianwen-2 mission, the probe will investigate an asteroid called 469219 Kamo'oalewa, which orbits the Sun at a distance relatively close to Earth. Hence, statement 1 is not correct and statement 2 is correct. — If the mission is successful, China will join a small group of countries, including the United States and Japan, that have successfully sampled and returned asteroids to Earth. — Kamo'oalewa was found in 2016 by the Pan-STARRS 1 asteroid survey telescope on Haleakalā, Hawaii. It is one of only seven asteroids classified as quasi-satellites of Earth, which are satellites that circle the Sun but are gravitationally impacted by the planet due to their proximity. Therefore, option (b) is the correct answer. Consider the following statements: Statement 1: When the leaves from trees near a volcano turn greener, it is indicative of the fact that it is active and is about to erupt soon. Statement 2: Greener trees detect early signs of an active volcano by tracing the volcanic carbon dioxide (CO₂). Which one of the following is correct in respect of the above statements? (a) Both Statement 1 and Statement 2 are correct and Statement 2 is the correct explanation for Statement 1. (b) Both Statement 1 and Statement 2 are correct and Statement 2 is not the correct explanation for Statement 1. (c) Statement 1 is correct but Statement 2 is incorrect. (d) Statement 1 is incorrect but Statement 2 is correct. Explanation — NASA has created a new method of observing volcano patterns by tracking tree health using satellite photos. In collaboration with the Smithsonian Institution's AVUELO (Airborne Validation Unified Experiment: Land to Ocean), space agency experts say they can detect early signals of volcanic eruptions by analysing the changing colours of tree leaves. — The scientists discovered that when the leaves of trees surrounding a volcano turn greener, it indicates that the volcano is active and may erupt soon. Tracing the CO₂ consumption patterns of nearby trees can help detect early indicators of an active volcano. Hence, statements 1 and 2 are correct. — Research indicates that rising magma beneath Earth's surface emits CO₂ and SO₂ into the atmosphere. While detecting sulphur dioxide in the atmosphere is relatively simple, scientists suggest that traces of volcanically produced carbon dioxide are far more difficult to identify. CO₂ can be difficult to identify from other gases in the atmosphere. Both Statement 1 and Statement 2 are correct and Statement 2 is the correct explanation for Statement 1. Therefore, option (a) is the correct answer. Rocket propulsion relies on: 1. Newton's first law of motion 2. Newton's third law of motion. 3. Archimedes' Principle Select the correct answer using the codes given below: (a) 1 only (b) 2 only (c) 1 and 2 only (d) 2 and 3 only Explanation — Ballistic missiles are still used today, but they are far more complex. A ballistic missile is one that is only powered during the initial stages of its flight. After that, it follows a parabolic course, much like a pebble tossed into the air, but quicker and farther. — A typical intercontinental ballistic missile (ICBM) may reach altitudes of more than 1,000 kilometres and speeds of Mach 20 (20 times the speed of sound). Once launched, they are virtually impossible to intercept. But pure ballistic routes are predictable — which is both their strength and weakness. — Guided missiles are equipped with sensors (such as radar, infrared, or GPS) and control systems (gyroscopes, fins, and internal thrusters) that allow them to direct themselves while in flight. — Rocket propulsion follows Newton's Third Law, which states that for every action, there is an equal and opposite reaction. The rocket moves ahead as it burns fuel and expels petrol from the back. The major problem isn't only travelling fast; it's also regulating flight at high speeds. Therefore, option (b) is the correct answer. With reference to the planet Uranus, consider the following statements: 1. It is pale blue-green in colour because its atmosphere absorbs the red wavelengths of sunlight. 2. As per the study, Uranus' atmosphere is primarily composed of hydrogen and helium, along with small amounts of methane. 3. Uranus' axis of rotation is nearly parallel to its orbital plane. How many of the statements given above are correct? (a) Only one (b) only two (c) All three (d) None Explanation — Uranus, the seventh planet from the Sun, owes its pale blue-green colour to its atmosphere that absorbs the red wavelengths of sunlight, according to a new study. Hence, statement 1 is correct. — The Hubble photos of Uranus were obtained between 2002 and 2022. According to the study, Uranus' atmosphere is mostly made of hydrogen and helium, with trace amounts of methane, water, and ammonia. Hence, statement 2 is correct. — Uranus is situated between Saturn and Neptune. Uranus, the seventh planet from the Sun, is one of the least studied planets in our solar system, which is why the latest research study could be significant. Scientists who wrote the paper also supplied additional information regarding seasonal changes on the planet. Unlike other planets, Uranus' axis of rotation is almost parallel to its orbital plane. Hence, statement 3 is correct. Therefore, option (c) is the correct answer. Sorry for the inconvenience caused. All remaining questions and answers are correctly marked. Daily Subject-wise quiz — History, Culture, and Social Issues (Week 112) Daily subject-wise quiz — Polity and Governance (Week 112) Daily subject-wise quiz — Science and Technology (Week 111) Daily subject-wise quiz — Economy (Week 111) Daily subject-wise quiz — Environment and Geography (Week 111) Daily subject-wise quiz – International Relations (Week 111) Subscribe to our UPSC newsletter and stay updated with the news cues from the past week. Stay updated with the latest UPSC articles by joining our Telegram channel – IndianExpress UPSC Hub, and follow us on Instagram and X.
