Scientists are seeing promising early results from the first studies testing gene editing for painful, inherited blood disorders that plague millions worldwide, especially Black people.Doctors hope the one-time treatment, which involves permanently altering DNA in blood cells with a tool called CRISPR, may treat and possibly cure sickle cell disease and beta thalassemia.Partial results were presented Saturday at an American Society of Hematology conference and some were published by The New England Journal of Medicine.Doctors described 10 patients who were at least several months removed from their treatment. All no longer needed regular blood transfusions and were free from the pain that plagued their lives before.Victoria Gray, the first patient in the sickle cell study, had long suffered bouts of severe pain that often sent her to the hospital.”I had aching pains, sharp pains, burning pains, you name it. That’s all I’ve known my entire life,” said Gray, 35, of Forest, Mississippi. “I was hurting everywhere my blood flowed.”Since her treatment a year ago, Gray has weaned herself from pain medications she depended on to manage her symptoms.”It’s something I prayed for my whole life,” she said. “I pray everyone has the same results I did.”Who’s affectedSickle cell affects millions, mostly Black people. Beta thalassemia strikes about one in 100,000 people. The only cure now is a bone marrow transplant from a closely matched donor without the disease, like a sibling, which most people don’t have.Both diseases involve mutations in a gene for hemoglobin, the substance in red blood cells that carries oxygen throughout the body.In sickle cell, defective hemoglobin leads to deformed, crescent-shaped blood cells that don’t carry oxygen well. They can stick together and clog small vessels, causing pain, organ damage and strokes.Those with beta thalassemia don’t have enough normal hemoglobin and suffer anemia, fatigue, shortness of breath and other symptoms. Severe cases require transfusions every two to five weeks.The treatment studied attacks the problem at its genetic roots.In the womb, fetuses make a special type of hemoglobin. After birth, when babies breathe on their own, a gene is activated that instructs cells to switch and make an adult form of hemoglobin instead. The adult hemoglobin is what’s defective in people with one of these diseases. The CRISPR editing aims to cut out the switching gene.”What we are doing is turning that switch back off and making the cells think they are back in utero, basically,” so they make fetal hemoglobin again, said one study leader, Dr. Haydar Frangoul of the Sarah Cannon Research Institute in Nashville.Switching gene targetedThe treatment involves removing stem cells from the patient’s blood, then using CRISPR in a lab to knock out the switching gene. Patients are given strong medicines to kill off their other, flawed blood-producing cells. Then they are given back their own lab-altered stem cells.Saturday’s results were those for the first 10 patients, seven with beta thalassemia and three with sickle cell. The two studies in Europe and the United States are ongoing and will enroll 45 patients each.Tests so far suggest the gene editing is working as desired with no unintended effects, Frangoul said.”The preliminary results are extremely encouraging,” he said.The study was sponsored by the therapy’s makers — CRISPR Therapeutics, with headquarters in Zug, Switzerland, and Massachusetts-based Vertex Pharmaceuticals. Some study leaders consult for the companies.Separately, Dr. David Williams of Harvard-affiliated Boston Children’s Hospital gave partial results from a study testing a novel type of gene therapy that also seeks to restore fetal hemoglobin production for those with sickle cell.Six patients including one as young as 7 were given the treatment, in which some of their blood stem cells were removed and altered in the lab to muffle the hemoglobin switching gene. None have had pain crises, five of the six no longer need transfusions and all have near-normal hemoglobin, he reported at the conference and in the medical journal.Government grants paid for the work. Williams is named on a patent for the therapy, which Boston Children’s has licensed to Bluebird Bio Inc. of Cambridge, Massachusetts. The company provided the therapy for the study, which will enroll 10 people in all to establish safety. A larger study to test effectiveness is planned.Williams, who was not involved in Frangoul’s study, said it “validates this approach” of targeting the hemoglobin switching gene to tackle sickle cell.
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Day: December 5, 2020
Japan’s space agency said its helicopter search team has spotted a capsule carrying asteroid samples that could explain the origin of life. It landed on a remote area in southern Australia as planned Sunday.Hayabusa2 successfully released the small capsule Saturday and sent it toward Earth to deliver samples from the distant asteroid that could provide clues to the origin of the solar system and life on our planet, the Japan Aerospace Exploration Agency said.Early Sunday, the capsule briefly turned into a fireball as it reentered the atmosphere 120 kilometers (75 miles) above Earth. About 10 kilometers (6 miles) above ground, a parachute opened to slow its fall, and beacon signals were transmitted to indicate its location.”It was great. … It was a beautiful fireball, and I was so impressed,” said JAXA’s Hayabusa2 project manager, Yuichi Tsuda, as he celebrated the successful capsule return and safe landing from a command center in Sagamihara, near Tokyo. “I’ve waited for this day for six years.”The capsule landed safely in a remote, sparsely populated area of Woomera, Australia, said JAXA official Akitaka Kishi.Searchers spot capsuleAbout two hours after the capsule’s reentry, JAXA said its helicopter search team found the capsule in the planned landing area. A retrieval of the pan-shaped capsule, about 40 centimeters (15 inches) in diameter, will start after the sunrise, Kishi said.The fireball could be seen from the International Space Station. Japanese astronaut Soichi Noguchi, who is now on a six-month mission there, tweeted: “Just spotted #hayabusa2 from #ISS! Unfortunately not bright enough for handheld camera, but enjoyed watching capsule!”Hayabusa2 left the asteroid Ryugu, about 300 million kilometers (180 million miles) away, a year ago. After it released the capsule, it moved away from Earth to capture images of the capsule descending toward the planet as it set off on a new expedition to another distant asteroid.Australian National University space rock expert Trevor Ireland, who was in Woomera for the arrival of the capsule, said he expected the Ryugu samples to be similar to the meteorite that fell in Australia near Murchison in Victoria state more than 50 years ago.”The Murchison meteorite opened a window on the origin of organics on Earth because these rocks were found to contain simple amino acids as well as abundant water,” Ireland said. “We will examine whether Ryugu is a potential source of organic matter and water on Earth when the solar system was forming, and whether these still remain intact on the asteroid.”Scientists say they believe the samples, especially ones taken from under the asteroid’s surface, contain valuable data unaffected by space radiation and other environmental factors. They are particularly interested in analyzing organic materials in the samples.Clues about distributionJAXA hopes to find clues to how the materials are distributed in the solar system and are related to life on Earth. Yoshikawa, the mission manager, said 0.1 gram of the dust would be enough to carry out all planned research.For Hayabusa2, it’s not the end of the mission it started in 2014. It is now heading to a small asteroid called 1998KY26 on a journey slated to take 10 years one way, for possible research including finding ways to prevent meteorites from hitting Earth.So far, its mission has been fully successful. It touched down twice on Ryugu despite the asteroid’s extremely rocky surface, and successfully collected data and samples during the 1½ years it spent near Ryugu after arriving there in June 2018.In its first touchdown in February 2019, it collected surface dust samples. In a more challenging mission in July that year, it collected underground samples from the asteroid for the first time in space history after landing in a crater that it created earlier by blasting the asteroid’s surface.Asteroids, which orbit the sun but are much smaller than planets, are among the oldest objects in the solar system and therefore may help explain how Earth evolved.Ryugu in Japanese means “Dragon Palace,” the name of a sea-bottom castle in a Japanese folk tale.
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A Nigerian yellow fever outbreak detected early last month is worsening and causing many cases and deaths across five of the country’s 36 states. The World Health Organization says 530 suspected cases, including 172 deaths, have been reported in Delta, Enugu, Benue and Ebonyi states in southern Nigeria and Bauchi in the north. Bringing this epidemic under control is difficult because Nigeria is facing
many simultaneous outbreaks of other infectious diseases, including Lassa fever, vaccine-derived polio virus, measles, monkey pox and cholera. NigeriaWorld Health Organization spokesman Tarik Jasarevic says the northeast of the country is also facing a humanitarian crisis largely caused by Boko Haram militants.“The response is particularly challenging in a COVID-19 context because it requires an extraordinary amount of time and resources from the country’s health system,” said Jasarevic. “…National and state authorities are currently focused on the COVID-19 pandemic response, limiting the human resources required to conduct investigations and response activities for the yellow fever outbreaks.”
Nigeria has been battling successive yellow fever outbreaks since 2017. The deadly disease is caused by a virus spread through the bite of infected Aedes aegypti mosquitos, which bite during the day. Jasarevic says vaccination is of key importance in preventing outbreaks in high-risk countries.FILE – An Aedes aegypti mosquito.“In Nigeria, these efforts are based on the introduction of the yellow fever vaccine into the routine immunization program,” said Jasarevic. “This started in 2004, and also carrying preventive campaigns to rapidly increase protection. But still the population, entire population of Nigeria has not been protected.” The World Health Organization urges those in areas reporting yellow fever to avoid daytime mosquito bites, keep their home surroundings clean, and clear mosquito breeding areas. It also recommends that anyone visiting Nigeria who is at least 9 months old be vaccinated against yellow fever.
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Japan’s space agency said the Hayabusa2 spacecraft successfully separated a capsule and sent it toward Earth to deliver samples from a distant asteroid that could provide clues to the origin of the solar system and life on our planet.The Japan Aerospace Exploration Agency said the capsule successfully detached Saturday afternoon from 220,000 kilometers away in a challenging operation that required precision control. The capsule is now descending to land in a remote, sparsely populated area of Woomera, Australia, on Sunday.Hayabusa2 left the asteroid Ryugu a year ago. After releasing the capsule, it is now moving away from Earth to capture images of the capsule descending to the planet.Yuichi Tsuda, project manager at the space agency JAXA, stood up and raised his fists as everyone applauded the moment command center officials confirmed the successful separation of the capsule.Hayabusa2’s return with the world’s first asteroid subsurface samples comes weeks after NASA’s OSIRIS-REx spacecraft made a successful touch-and-go grab of surface samples from asteroid Bennu. China, meanwhile, announced this week its lunar lander collected underground samples and sealed them within the spacecraft for return to Earth, as nations compete in space missions.Many Hayabusa2 fans gathered to observe the moment of the capsule separation at public viewing events across the country, including one at the Tokyo Dome stadium.In the early hours of Sunday, the capsule, protected by a heat shield, will briefly turn into a fireball as it reenters the atmosphere 120 kilometers above Earth. At about 10 kilometers above ground, a parachute will open to slow its fall and beacon signals will be transmitted to indicate its location.JAXA staff have set up satellite dishes at several locations in the target area to receive the signals, while also preparing a marine radar, drones and helicopters to assist in the search and retrieval of the pan-shaped capsule, 40 centimeters in diameter.Australian National University space rock expert Trevor Ireland, who is in Woomera for the arrival of the capsule, said he expected the Ryugu samples to be similar to the meteorite that fell in Australia near Murchison in Victoria state more than 50 years ago.“The Murchison meteorite opened a window on the origin of organics on Earth because these rocks were found to contain simple amino acids as well as abundant water,” Ireland said, “We will examine whether Ryugu is a potential source of organic matter and water on Earth when the solar system was forming, and whether these still remain intact on the asteroid.”Scientists say they believe the samples, especially ones taken from under the asteroid’s surface, contain valuable data unaffected by space radiation and other environmental factors. They are particularly interested in analyzing organic materials in the samples.JAXA hopes to find clues as to how the materials are distributed in the solar system and are related to life on Earth.For Hayabusa2, it’s not the end of the mission it started in 2014. After dropping the capsule, it will return to space and head to another distant small asteroid called 1998KY26 on a journey slated to take 10 years one way, for a possible research including finding ways to prevent meteorites from hitting Earth.So far, its mission has been fully successful. It touched down twice on Ryugu despite its extremely rocky surface, and successfully collected data and samples during the 1½ years it spent near Ryugu after arriving there in June 2018.In its first touchdown in February 2019, it collected surface dust samples. In a more challenging mission in July that year, it collected underground samples from the asteroid for the first time in space history after landing in a crater that it created earlier by blasting the asteroid’s surface.Asteroids, which orbit the sun but are much smaller than planets, are among the oldest objects in the solar system and therefore may help explain how Earth evolved.Ryugu in Japanese means “Dragon Palace,” the name of a sea-bottom castle in a Japanese folk tale.
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Russia has begun its COVID-19 vaccination program. Seventy clinics in Moscow began inoculating people Saturday with the Sputnik-V COVID-19 shot, the city’s coronavirus task force said.The vaccine is being made available to health care workers, social workers and people who work in schools because they run the highest risk of exposure to the coronavirus. People over 60 are excluded from receiving the shot, media reports say.Russia’s vaccine is administered in two injections, with the second injection scheduled for three weeks after the first.Thousands of people have registered to receive the vaccine. It was not immediately clear, however, how much of the vaccine has been produced.Some scientists have questioned the efficacy of the Russian-manufactured vaccine because of its speedy appearance on the market. Russia has 2.4 million COVID infections and more than 42,000 deaths, according to Johns Hopkins. The California city of San Francisco and several Bay Area counties said Friday that they will begin imposing stay-at-home orders this weekend as part of their battle against the coronavirus.California Gov. Gavin Newsom said Thursday that the state was on the verge of imposing stay-at-home orders on a regional basis once intensive care units in the state’s five regions reached more than 85% capacity.San Francisco and the Bay Area counties, however, are not waiting for the hospital capacity threshold and are instead voluntarily opting into the state’s regional stay-at-home order.”We are in our worst surge yet of COVID-19. It is stressing health care systems across the state of California and taxing our health care workers,” Dr. Grant Colfax, San Francisco’s director of health, said Friday. “We need urgent intervention now if we want to be able to care for the sick in mid-to-late December. We do not want your parent, your spouse, your child, your grandparent or any loved one to be in need of help and our hospitals too overwhelmed to properly care for them.”FILE – California Street, usually filled with cable cars, is seen empty in San Francisco, Calif., on March 18, 2020, amid the COVID-19 pandemic.Starting Sunday night, the order will close all outdoor dining, public outdoor playgrounds, outdoor museums, zoos and aquariums, drive-in theaters, and open-air tour buses and boats. Pet grooming and electronics or shoe repair, considered low-contact retail, will be allowed to operate on a curbside-dropoff basis. All other retail, including grocery stores, will be allowed to operate only at 20% capacity.“We must do whatever is necessary in order to get the virus under control,” said San Francisco Mayor London Breed. “This is about protecting people’s lives.”The head of the World Health Organization said Friday that with a COVID-19 vaccine on the horizon, nations must start investing and preparing for the next pandemic.“Despite years of warnings, many countries were simply not ready for COVID-19,” WHO Director-General Tedros Adhanom Ghebreyesus told a special session of the U.N. General Assembly on the coronavirus. “Many mistakenly assumed their strong health systems would protect them.”He said countries that have dealt with recent coronaviruses, including SARS, or severe acute respiratory syndrome, and MERS, or Middle East respiratory syndrome, as well as other infectious diseases, have done better in containing COVID-19.“Now all countries must develop that same muscle memory and invest in the measures that will prevent, control and mitigate the next crisis,” Tedros said. “It is also clear the global system for preparedness needs attention.”FILE – A pedestrian walks past a sign advising mask-wearing during the coronavirus outbreak in San Francisco, Nov. 21, 2020.The WHO has come in for criticism from some countries for its handling of the pandemic after China reported the first cases early this year. U.S. President Donald Trump has been one of the most vocal critics, and on May 29 announced the United States would withdraw from the global health organization. President-elect Joe Biden has said he will reverse that decision when he takes office in January.The WHO chief stressed the need for rich and poor countries alike to have equal access to a COVID-19 vaccine, saying sharing science is not charity, but in the best interest of every nation. He also urged nations to radically rethink how they prioritize and view health if they want to avoid another crisis on this scale.“The pandemic has proven that a health crisis is not just a health crisis, it’s a social, economic, political and humanitarian crisis,” he said. “The risks of under investment in health have wide-ranging impacts, and so do the benefits of investing in health.”On Friday, Bahrain became the second country to approve emergency use of the Pfilzer-BioNTech coronavirus vaccine. Britain was the first nation to greenlight the vaccine.The challenge would be keeping the vaccine cold enough. It must be stored at temperatures around minus 70 degrees Celsius. Bahrain routinely registers summer temperatures of 40 degrees Celsius.Bahrain has already inoculated 6,000 people with a Chinese vaccine that uses a dead version of the virus. The Mideast nation has had nearly 88,000 cases of the coronavirus and almost 350 deaths, according to Johns Hopkins Coronavirus Resource Center.Global COVID-19 confirmed cases have surpassed 65 million with more than 1.5 million deaths. The U.S. continues to have the highest number of confirmed cases – more than 14 million so far — and nearly 279,000 deaths, according to Johns Hopkins University.
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U.S. President Donald Trump signed into law Friday a bill that lets U.S. justice officials pursue criminal penalties against those involved in doping conspiracies at international events involving American athletes, sponsors or broadcasters.The Rodchenkov Act, named after the whistleblower Grigory Rodchenkov who helped expose Russia’s state-sponsored doping, empowers prosecutors to seek fines of up to $1 million and jail terms of up to 10 years, as well restitution to victims.”(The law gives) the Department of Justice a powerful and unique set of tools to eradicate doping fraud and related criminal activities from international competitions,” said Rodchenkov’s lawyer, Jim Walden, according to Inside the Games.It is now up to the Justice Department to develop a robust program, cooperating with the U.S. Anti-Doping Agency and international law enforcement, to bring the guilty to justice and create zero tolerance for doping in sports, he added.The bill, passed unopposed by the U.S. Senate last month, was opposed by the International Olympic Committee, who have questioned why American professional and college athletes are exempt.The United States Anti-Doping Agency (USADA) said there was no need to include U.S. professional and college sports in the legislation as existing law allows their prosecution.The World Anti-Doping Agency also expressed concerns that the bill would destabilize the global anti-doping effort by extending U.S. jurisdiction beyond its own borders.”No nation has ever before asserted criminal jurisdiction over doping offenses that occurred outside its national borders — and for good reason,” the agency said last month when the bill passed the Senate.”WADA remains concerned that by unilaterally exerting U.S. criminal jurisdiction over all global doping activity, the Act will likely undermine clean sport by jeopardizing critical partnerships and cooperation between nations.”
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