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☆ Experimental gene therapy restores some vision in patients with inherited blindness
▼ + stars: | 2024-05-06 | by ( Jacqueline Howard | ) edition.cnn.com time to read: +16 min

“Now, post-surgery and post recovery, I am able to see in dimmer lighting with my left eye,” Cook said. A treatment that used CRISPR was found to be safe and efficacious in improving vision among a small sample of patients with inherited blindness in the Phase 1/2 clinical trial that Cook participated in. Months following the treatment, Cook was sitting with friends on a balcony that had Christmas lights wrapped around the railing. Courtesy Olivia CookBefore the treatment, Cook said that she sometimes could conceal the vision challenges she has had. Mass Eye and EarKalberer described the CRISPR treatment as “groundbreaking,” but warned it’s not a cure.

Persons: Olivia Cook, Cook, ”, ” Cook, “, I’ve, “ I’d, you’d, CRISPR, Eric Pierce, Brigham, “ We’re, ” Pierce, Jason Comander, ‘, Michael Kalberer, Kalberer, it’s, “ It’s, It’s, Pierce, Editas, We’re, Art Caplan, ” Caplan, —, Vlad Diaconita, ” Diaconita, Dr, Sanjay Gupta, Aliaa Abdelhakim Organizations: CNN, Missouri State University, New England, of Medicine, Mass, Harvard Medical School, Editas Medicine, Perelman School of Medicine, University of Pennsylvania, University of Michigan, University of Miami, Oregon Health & Science University, US Food and Drug Administration, CEP290, pharma, NYU Grossman School, Medicine’s Department of Population Health, Columbia University Vagelos College of Physicians and Surgeons, CNN Health Locations: Springfield, United States, CEP290

☆ Citi names 3 biotech stocks to play a growing $2.9 billion opportunity — giving one about 50% upside
▼ + stars: | 2024-05-01 | by ( Weizhen Tan | ) www.cnbc.com time to read: +2 min

The outlook is starting to look bright for biotech stocks, according to some. With markets now expecting the first rate cut to be in September rather than June or July, as previously thought, biotech stocks could start to do well. Biotech encompasses many different areas, but Citi has identified one with a $2.9 billion market — which it says is set for even more growth. It gave CSL a price target of $305, or nearly 11% potential upside. It gave Intellia a price target of $31, or 49% potential upside.

Persons: Morgan Stanley, Garadacimab, Ionis, Citi Organizations: Biotech, Citi, Ionis Pharmaceuticals, Intellia Therapeutics, CSL, Intellia

☆ UK regulators approve medical treatment involving CRISPR gene editing in world first
▼ + stars: | 2023-11-16 | by ( Katie Hunt | ) edition.cnn.com time to read: +4 min

CNN —The United Kingdom has become the first country to give regulatory approval to a medical treatment involving the revolutionary CRISPR gene editing tool. The treatment, made by Vertex Pharmaceuticals, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a lab. “Modifying the stem cells from the bone marrow of the patient avoids the problems associated with immune compatibility, i.e. The release from the MHRA did not say how much the treatment would cost, but it’s likely to be expensive. CRISPR-Cas9 has had a major impact on biomedical research, clinical medicine and agriculture and is widely used in labs around the world.

Persons: —, ”, Julian Beach, “, — Emmanuelle Charpentier, Jennifer A, Doudna —, Casgevy, Alena Pance, ” Pance, Cas9, Jiankui Organizations: CNN, country’s Medicines, Healthcare, Agency, Beta, Vertex Pharmaceuticals, US Food and Drug Administration, University of Hertfordshire, Science Media Centre Locations: United Kingdom, South Asian

☆ What the Nobel Prizes get wrong about science
▼ + stars: | 2023-09-29 | by ( Katie Hunt | ) edition.cnn.com time to read: +9 min

Peter Brzezinski, the secretary of the committee for the Nobel chemistry prize, said there were no plans to change the rule. He said the Nobel Prize committees, at least for science prizes, are “innately conservative.”DiversityOther criticism leveled at the Nobel Prizes includes the lack of diversity among winners. Of course, these flaws and gaps only matter because the Nobels are far better known than other science prizes, Rees added. The Nobel Prize in physiology or medicine will be announced on Monday, followed by the physics prize on Tuesday and the Nobel Prize in chemistry on Wednesday. The Nobel Prize for literature and the Nobel Peace Prize will be announced on Thursday and Friday, respectively.

Persons: Alfred Nobel, Martin Rees, Rees, ”, Jonathan Nackstrand, Rainer Weiss, Barry Barish, Kip Thorne, David Pendlebury, “ Nobel, ” Pendlebury, Nobel’s, Peter Brzezinski, “, ” Brzezinski, John Jumper, AlphaFold, Lasker, Pendlebury, Emmanuelle Charpentier, Jennifer Doudna, it’s, Carolyn Bertozzi, Andrea Ghez, Naomi Oreskes, Henry Charles Lea, ” Rees Organizations: CNN, Royal Society, Getty, Clarivate’s Institute for Scientific, Nobel Foundation, Academy, Google, Harvard University Locations: Swedish, AFP, Stockholm

☆ Bayer claims early lead in Parkinson's stem cell therapy
▼ + stars: | 2023-06-28 | by ( Ludwig Burger | ) www.reuters.com time to read: +3 min

Some of this work is done by Britain's' Cambridge University, South Korea's Bundang CHA Hospital, International Stem Cell Corp's (ISCO.PK) Cyto Therapeutics in Australia, the Chinese Academy of Sciences, Harvard University and Japan's Kyoto University Hospital. For BlueRock's experimental therapy, researchers took induced pluripotent stem cells, which are modified to regain the ability to form any type of specialised tissue, and transformed them into dopamine-producing nerve cells. When surgically implanted into the brain of a person with Parkinson's disease, the therapeutic cells are designed to restore neural networks destroyed by the disease. Initial trial results showed the cells multiplied and started making dopamine, an important brain signalling molecule which is lacking in Parkinson's patients. Parkinson's, for which there is no cure and which affects more than 10 million people worldwide, causes progressive brain damage.

Persons: Wolfgang Rattay, Bayer, BlueRock, Britain's, Jennifer Doudna, Ludwig Burger, Miranda Murray, Mark Potter Organizations: Bayer AG, REUTERS, Bayer, Cambridge University, South Korea's, CHA Hospital, Cyto Therapeutics, Chinese Academy of Sciences, Harvard University, Japan's Kyoto University Hospital, BlueRock Therapeutics, Mammoth Biosciences, Thomson Locations: Leverkusen, Germany, FRANKFURT, Australia, San Francisco Bay

☆ These innovation stocks owned by Cathie Wood have double-digit upside, according to analysts
▼ + stars: | 2023-02-06 | by ( Yun Li | ) www.cnbc.com time to read: +2 min

Cathie Wood just scored her best month ever as her innovation stocks staged a big comeback, and some of her darlings still have double-digit upside ahead, according to Wall Street analysts. Wood's flagship Ark Innovation ETF (ARKK) jumped 27.8% in January alone, notching its strongest month since its inception in 2014 . We looked at companies with at least five analysts covering them, and these names below all have more than 10% upside in the next 12 months, according to FactSet. Wall Street analysts are most bullish on Ginkgo Bioworks , seeing the stock rally more than 140% in the next 12 months. Other biotech names in the space that analysts love are Verve Therapeutics , CRISPR Therapeutics , Beam Therapeutics and Twist Bioscience.

☆ A tiny biotech just suffered a safety scare in a gene-editing trial, raising 3 big questions for the entire CRISPR field
▼ + stars: | 2023-01-06 | by ( Andrew Dunn | ) www.businessinsider.com time to read: +5 min

The biotech halted the trial while it investigates the case, which may impact other CRISPR companies. The small biotech company Graphite Bio said Thursday afternoon that it paused a study testing an experimental gene-editing therapy after the first treated patient suffered a serious side effect. The safety scare raises more questions than answers as Graphite's scientists investigate what exactly may have caused the side effect. Lehrer said it's too early to say whether this safety scare could have implications for the broader gene-editing field. Its next-closest product, a gene-editing therapy for beta-thalassemia, isn't expected to start initial human testing until 2024 at the earliest.

☆ How a gene-editing breakthrough from a Harvard lab saved the life of a girl with leukemia
▼ + stars: | 2022-12-19 | by ( Andrew Dunn | ) www.businessinsider.com time to read: +6 min

A 13-year-old girl was the world's first patient to get a cell therapy called base editing in May. Base editing is a new gene-editing technology that could lead to cures for many diseases. Alyssa, 13, was the first-ever patient to receive a base-edited cell therapy after enrolling in a clinical trial in May. Base editing allows scientists to make ultraprecise changes to single letters of DNA in cells. Alyssa, a teenager in Leicester, England, received a base-edited cell therapy to treat her leukemia.

☆ We went inside the lab at Vertex, a company that dodged the market downturn to become one of the best-performing biotechs of 2022
▼ + stars: | 2022-09-21 | by ( Andrew Dunn | ) www.businessinsider.com time to read: +10 min

A mosaic of human cells inside Vertex Pharmaceuticals' cell and gene-therapy laboratory. The four-story, 267,000-square-foot space is home to Vertex's cell and gene-therapy unit, with about 375 people now residing in the modern-industrial building. That has led to going after unrelated diseases like sickle cell, diabetes, muscular dystrophy, kidney disease, and pain. For instance, Vertex's approach to sickle cell is led by its CRISPR gene-editing program. Vertex PharmaceuticalsThe next test of Vertex's strategy will be an experimental treatment called exa-cel, its CRISPR gene-editing program for sickle cell.

☆ Una dintre problemele acestui secol ar putea fi rezolvată cu ajutorul ingineriei genetice; Tehnica este însă controversată
▼ + stars: | 2021-07-04 | by ( ) www.jurnal.md time to read: +4 min

Sursa foto: ProfimediaUna dintre problemele acestui secol ar putea fi rezolvată cu ajutorul ingineriei genetice; Tehnica este însă controversatăSoluția la una dintre problemele acestui secol - rezistența tot mai mare a bacteriilor la antibiotice - ar putea fi rezolvată cu ajutorul ingineriei genetice. Ar putea deveni o armă redutabilă de luptă împotriva superbacteriilor, însă unele consecințe ar putea fi imprevizibile și mai putin sănătoase. Dezvoltarea de noi antibiotice a devenit unul dintre obiectivele cele mai presante ale timpului nostru. Antibioticele au fost folosite pentru trata numeroase infecții începând cu 1940, când penicilina a fost dezvoltată dintr-un tip de ciupercă numit Penicillium. În ciuda entuziasmului, există și îngrijorări: una dintre ele ia în calcul riscurile necunoscute pe care le implică modificarea artificială a virusurilor și a bacteriilor.

Persons: David Edgell, Jason Micklefield Organizations: New Scientist, Universitatea de Vest, Universitatea din Manchester, Universitatea din, Manchester Locations: New, Canada, Universitatea din Manchester, Universitatea din Cambridge

☆ Разработана генная терапия, которая может замедлить процесс старения
▼ + stars: | 2021-01-21 | by ( Mld Media Srl | ) noi.md time to read: +1 min

Ученые из трех китайских исследовательских институтов объединили свои усилия, чтобы выявить новые гены, которые отвечают за старение человеческого организма. При этом они обнаружили еще около полусотни генов, которые могут запустить процесс омоложения организма, если их «отключить». Оказалось, что в период, когда прослеживается экспрессия этого гена, клетки стремительно начинают стареть и, наоборот, процесс замедляется при его ослаблении. Китайские ученые считают, что при условии инактивации KAT7, стволовые клетки, уже затронутые процессом старения, начнут омолаживаться. Однако для того, чтобы можно было без опасений использовать данный метод лечения, необходимо провести еще множество дополнительных экспериментов.

☆ Cercetătorii chinezi au dezvoltat o terapie genică care ar putea întârzia procesul de îmbătrânire
▼ + stars: | 2021-01-20 | by ( Publika.Md - Aici Sunt Știrile | ) www.publika.md time to read: +2 min

O echipă de cercetători din Beijing a dezvoltat o nouă terapie genică ce poate inversa unele dintre efectele îmbătrânirii la şoareci şi le poate extinde durata de viaţă. Astfel, reușita oamenilor de știință ar putea contribui în viitor la obținerea unui tratament similar pentru oameni, scrie Reuters, citat de Agerpres. Metoda a fost detaliată într-o lucrare științifică și implică dezactivarea unei gene numite kat7, despre care oamenii de ştiinţă au descoperit că joacă un rol esenţial în procesul de îmbătrânire celulară. „După şase-opt luni, aceşti şoareci prezintă o îmbunătăţire generală a aspectului şi a puterii de apucare şi, cel mai important, au o durată de viaţă extinsă cu aproximativ 25%”, a declarat Qu. În cele din urmă, sperăm că putem găsi o modalitate de a întârzia îmbătrânirea, chiar şi cu un procent foarte mic... în viitor”, a mai adăugat Qu.

Persons: Jing, cercetărotii Organizations: Agerpres, Academiei, Ştiinţe, Kat7 Locations: Beijing, China

☆ Cum este să fii testat cu vaccinul rusesc Sputnik V — Moldova.org
▼ + stars: | 2020-10-21 | by ( Diana Preașcă | Tatiana Beghiu | ) www.moldova.org time to read: +20 min

Primul om care a primit Premiul Nobel pentru Pace a fost fondatorul Crucii Roșii, elvețianul Henri Dunant în 1901. Din 1901, premiul a fost acordat unui număr total de 213 de cercetători, dintre care doar trei au fost femei. Mecanismul a fost numit Crispr/Cas9 și a fost supranumit „foarfecele genetic”. Este angajată a Institutului de Medicină Howard Hughes și membră Academiei de Științe și a Academiei de Medicină din SUA. Din 1901, premiul a fost acordat, în total, de 111 ori, iar 184 de oameni de știință au devenit laureați ai acestuia.

Persons: Nobel, Pace, Antonio, PAM, SUA Dwight D . Eisenhower, Henri Dunant, Theodore Roosevelt, Martin Luther King, german Willie Brandt, SUA Henry Kissinger, Teresa, SUA Barack Obama, Roger Penrose, Reinhard Genzel, Andrea Ghez, Max Planck, . Penrose, Albert Einstein, Sir Roger Penrose, Stephen Hawking, Wilhelm, Nielson Bohr, Marie Curie, Pierre Curie, Antoine Henri Becquerel, franceze Emmanuelle Charpentier, Jennifer Doudna, Emmanuelle Charpentier, Medicină Howard Hughes, Jacobus, van’t, Maria, Curie, Harvey J ., Michael Houghton, Charles M . Rice, virusologul Harvey J ., Chiron, Houghton, american Charles M . Rice, Louis, Emil Adolf von Bering, Ronald Ross, Robert Koch, Ivan Pavlov, Frederick Banting, John McLeod, Alexander Fleming, Ernest Chain, Howard Florey, Louise Glück, Glück, Anders Ohlsson, Sarah Lawrence, Rudyard Kipling, Doris Lessing, Bob Dylan ., Svetlana Aleksievici, Paul R ., Robert B . Wilson, Stanford . Robert B . Wilson, Paul R . Milgrom, Milgrom, Wilson, Alfred Nobel, Jan Tinbergen, Ragnar Frisch Organizations: Universitatea din, Națiunilor Unite, ONU, Organizația, Organizația ONU, Crucii Roșii, Regale, franceze, Science, Academiei, Științe, Academiei Naționale, Medicină, Academiei de Științe, Academiei de Medicină, maimuțelor, Universitatea St, Organizației Mondiale a Sănătății, Academia Suedeză, Nobel, Academia suedeză, Universității Columbia ., Academiei Americane, Academiei Poeților, Universitatea Yale ., Universitatea Stanford, Stanford ., Universității Harvard, Stanford Business School, Banca Centrală, Academiei Regale Locations: Stockholm, Universitatea din Oslo, Roma, ONU, Republica Democrată Congo, Nigeriei, Sudanul de Sud, Siria, Yemen, german, SUA, Universitatea din Oxford, Germania, Universitatea din California, Berkeley, Los Angeles, Regatului Unit, Franța, Olanda, Universitatea din Alberta, Canada, american, Washington, americană, New York, maghiară, Yale, Boston, Iowa, engleză

☆ Cine sunt laureații Premiului Nobel 2020 și ce știm despre ei — Moldova.org
▼ + stars: | 2020-10-13 | by ( Diana Preașcă | Tatiana Beghiu | ) www.moldova.org time to read: +20 min

La Stockholm, ceremonia de premiere pentru câștigătorii Premiului Nobel și banchetul de gală de la primărie au fost anulate. Emmanuelle Charpentier (51 ani) și Jennifer Doudna (56 de ani) sunt a șasea și respectiv a șaptea câștigătoare ale Premiului Nobel pentru Chimie din istorie. Mecanismul a fost numit Crispr/Cas9 și a fost supranumit „foarfecele genetic”. Este angajată a Institutului de Medicină Howard Hughes și membră Academiei de Științe și a Academiei de Medicină din SUA. Premiul Nobel pentru MedicinăPremiul Nobel pentru Medicină le-a revenit în 2020 cercetătorilor Harvey J.

☆ Зрительный нерв впервые восстановили с помощью генной терапии
▼ + stars: | 2020-10-06 | by ( ) point.md time to read: +3 min

Молекулярные биологи придумали, как можно стимулировать восстановление зрительного нерва, с помощью которого в мозг попадает информация о том, что видят наши глаза. Статью с результатами исследования опубликовал научный журнал Nature Communications, передает tass.ru"С помощью генной терапии мы доставили белок протрудин в поврежденную часть нерва. Другие клетки, в том числе зрительный нерв, участвуют в передаче этих сигналов в центры зрения мозга. Ученые пытаются исправить подобные дефекты как с помощью стволовых клеток, которые могут превращаться в "заготовки" клеток сетчатки, так и генной терапии, которая "перепрограммирует" часть выживших клеток, заставляя их расти, превращаться в рецепторы или выполнять другие функции. Убедившись в этом, ученые проследили, как генная терапия действует на поврежденный зрительный нерв и сетчатку нескольких крыс.

Persons: Веселина Петрова, Петров Organizations: Nature Communications, Кембриджский университет Locations: Великобритания, США

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