BacktoCricaddict Posted May 10, 2024 Author Posted May 10, 2024 https://www.cuh.nhs.uk/news/baby-born-deaf-can-hear-after-breakthrough-gene-therapy/ Baby born deaf can hear after breakthrough gene therapy Baby girl born deaf can hear unaided for the first time, after receiving ground-breaking gene therapy when she was eleven months old at Addenbrooke’s Hospital in Cambridge. Opal Sandy from Oxfordshire is the first patient treated in a global gene therapy trial, which shows “mind-blowing” results. She is the first British patient in the world and the youngest child to receive this type of treatment. Opal was born completely deaf because of a rare genetic condition, auditory neuropathy, caused by the disruption of nerve impulses travelling from the inner ear to the brain. Within four weeks of having the gene therapy infusion to her right ear, Opal responded to sound, even with the cochlear implant in her left ear switched off. Auditory neuropathy can be due to a variation in a single gene, known as the OTOF gene. The gene produces a protein called otoferlin, needed to allow the inner hair cells in the ear to communicate with the hearing nerve. Approximately 20,000 people across the UK, Germany, France, Spain, Italy and UK and are deaf due to a mutation in the OTOF gene. Children with a variation in the OTOF gene often pass the newborn screening, as the hair cells are working, but they are not talking to the nerve. It means this hearing loss is not commonly detected until children are 2 or 3 years of age – when a delay in speech is likely to be noticed. Professor Manohar Bance added: “We have a short time frame to intervene because of the rapid pace of brain development at this age. Delays in the diagnosis can also cause confusion for families as the many reasons for delayed speech and late intervention can impact a children’s development.” Tillu 1
Real McCoy Posted May 12, 2024 Posted May 12, 2024 mRNA vaccines are Gene Therapy mRNA vaccines side effects 5 shots of mRNA vaccines result in this
BacktoCricaddict Posted May 12, 2024 Author Posted May 12, 2024 On 3/28/2024 at 7:17 PM, BacktoCricaddict said: Xenotransplants (organ transplants from other species to humans) - and sometimes even human-to-human transplants - can be very risky due to a violent immune rejection response in our bodies. For this reason, compatible kidney donors are so difficult to find and 1000s of patients die each year without the opportunity to get a transplant. The following research may provide a solution: -------------------------------------------------------------------------------------- https://www.massgeneral.org/news/press-release/worlds-first-genetically-edited-pig-kidney-transplant-into-living-recipient Today, Massachusetts General Hospital (MGH), a founding member of the Mass General Brigham health care system, announced the world’s first successful transplant of a genetically-edited pig (porcine) kidney into a 62-year-old man living with end-stage kidney disease (ESKD). The patient, Mr. Richard ‘Rick’ Slayman of Weymouth, Mass., is recovering well at MGH and is expected to be discharged soon. “The real hero today is the patient, Mr. Slayman, as the success of this pioneering surgery, once deemed unimaginable, would not have been possible without his courage and willingness to embark on a journey into uncharted medical territory. “The success of this transplant is the culmination of efforts by thousands of scientists and physicians over several decades. We are privileged to have played a significant role in this milestone. Our hope is that this transplant approach will offer a lifeline to millions of patients worldwide who are suffering from kidney failure,” Kawai said. The pig kidney was provided by eGenesis of Cambridge, Mass., from a pig donor that was genetically-edited using CRISPR-Cas9 technology to remove harmful pig genes and add certain human genes to improve its compatibility with humans. Additionally, scientists inactivated porcine endogenous retroviruses in the pig donor to eliminate any risk of infection in humans. Over the past five years, MGH and eGenesis have conducted extensive collaborative research, with the findings published in Nature in 2023. ---------------------------------------------------------------------------------------- Ethical concerns regarding taking another mammal's life to save a human's life may be raised (and will make for interesting debate), but if the patient was oneself or one's loved one, I'd imagine there would be no dilemma. Sadly, this patient didn't survive for more than 2 months. Xenotransplantation of kidneys has a long way to go before it can supplement or replace human transplants which have a 90% 12-month survival rate. The challenge with human transplants is finding a match. https://www.statnews.com/2024/05/12/xenotransplantation-crispr-pig-kidney-transplant-patient-dies/ Prakat 1
diga Posted May 12, 2024 Posted May 12, 2024 @BacktoCricaddict We dont hear much on HIV/AIDS nowadays.. why
Vijy Posted May 13, 2024 Posted May 13, 2024 (edited) 2 hours ago, BacktoCricaddict said: Sadly, this patient didn't survive for more than 2 months. Xenotransplantation of kidneys has a long way to go before it can supplement or replace human transplants which have a 90% 12-month survival rate. The challenge with human transplants is finding a match. https://www.statnews.com/2024/05/12/xenotransplantation-crispr-pig-kidney-transplant-patient-dies/ yes, sad news regd the pig-man chimera. long way to go still Edited May 13, 2024 by Vijy
Real McCoy Posted May 13, 2024 Posted May 13, 2024 9 hours ago, diga said: @BacktoCricaddict We dont hear much on HIV/AIDS nowadays.. why the disease has a new strain called vaccine acquired auto immune deficiency syndrome https://pubmed.ncbi.nlm.nih.gov/34957554/ diga 1
diga Posted May 13, 2024 Posted May 13, 2024 4 hours ago, Real McCoy said: the disease has a new strain called vaccine acquired auto immune deficiency syndrome https://pubmed.ncbi.nlm.nih.gov/34957554/ Hmm.. its scary that vaccination has given rise to immune deficiency. As you or someone mentioned, all I can hope is that I got a placebo.... But still the question remains... we dont see many news articles on AIDS/HIV compared to 2000s. Is it on the vane? Real McCoy 1
Real McCoy Posted May 13, 2024 Posted May 13, 2024 1 hour ago, diga said: Hmm.. its scary that vaccination has given rise to immune deficiency. As you or someone mentioned, all I can hope is that I got a placebo.... But still the question remains... we dont see many news articles on AIDS/HIV compared to 2000s. Is it on the vane? Maybe its not that scary anymore to the american public with the arrival of coronavirus. With lockdowns in place, everyone was glued into their TVs and social media regadring news of the virus. People went into panickstan. It was surreal. Who knows AIDS may have been a precursor to the recent epidemic. Some people took vaccination (Is it the heptatitis injection?) that time too because they were more prone to illicit sex.
BacktoCricaddict Posted May 13, 2024 Author Posted May 13, 2024 (edited) On 5/12/2024 at 7:59 PM, diga said: @BacktoCricaddict We dont hear much on HIV/AIDS nowadays.. why Over the past 2.5 decades, highly effective anti-retroviral therapies (HAART) have been developed, resulting in a drastic drop in mortality rates, viral load, and new infections. Here is some mortality data. It is not over by any means, but it is definitely on the wane. Nowadays, even Pre-infection preventative drug therapies are being used to successfully keep transmission down. I think the reason the media is not covering it much - and this is unfortunate - is because the media is acting like it is over based on the current mortality rates in the US, which are very low compared to their peak in 1995-97. https://www.cdc.gov/hiv/pdf/library/slidesets/cdc-hiv-surveillance-mortality-2019.pdf. In other countries too, especially Sub-Saharan Africa, it has improved a lot, but is still quite bad from an absolute numbers perspective. But since it is not rampant in the US, media has no interest. Also note that vaccine development has been very difficult because HIV has turned out to be extremely tricky to work with. Edited May 18, 2024 by BacktoCricaddict diga and Vijy 2
BacktoCricaddict Posted May 18, 2024 Author Posted May 18, 2024 On 5/12/2024 at 9:14 PM, Vijy said: yes, sad news regd the pig-man chimera. long way to go still Here are excerpts from a good article about the challenges ahead. https://www.nature.com/articles/d41586-024-01453-2 Quote The three procedures offered hope to desperately ill people who had run out of options. And researchers say that they have learnt valuable lessons from the first pig-organ transplants into humans, on topics ranging from the types of medication that recipients need to the amount of testing that pig organs must undergo. “This is not an insolvable problem,” Montgomery says. “I’m encouraged that we’re as far along as we are.” ---------------------------------------- The use of organs from other species in humans, called xenotransplantation, has long been a dream of surgeons because of the chronic shortage of suitable human organs. Researchers have homed in on pigs as a donor species, in part because their organs’ size and anatomy resemble those of humans. Data from non-human primates that have received pig organs are promising: a study1 published in 2023 reported that five monkeys each survived for more than one year after receiving transplanted pig kidneys. ------------------------------- In the weeks before he died, Bennett had an infection, so physicians gave him an immune-boosting therapy made up of pooled antibodies from thousands of donors. Scientists later found that some of the antibodies had reacted to the pig organ2, meaning that the treatment could have exacerbated Bennett’s condition. Since then, Mohiuddin has worked with local blood banks to develop ways to screen for reactive antibodies. Another possible explanation for Bennett’s limited survival is a latent infection of the transplanted heart with a pathogen called porcine cytomegalovirus, which might have been activated and then harmed the heart. The virus was found in the organ after Bennett’s death but was missed by tests before the transplant, signalling that more sensitive tests must be used to screen organs, Mohiuddin says. --------------------------------- In the fourth and latest xenotransplant in a live person, Montgomery and his team tried a new approach using the thymus, an immune-related organ that could help teach the recipient’s immune system to recognize the pig organ. They grafted the source pig’s thymus to the kidney and then transplanted both into 54-year-old Lisa Pisano on 12 April. They used a pig with only a single genetic modification, which could make scaling up the production of pig organs easier, Montgomery says. Pisano remains in stable condition in hospital, he adds. ----------------------- There is still much more to be learnt, he says. In a forthcoming study and in one published today in Nature Medicine3, Montgomery and his colleagues analysed tissue samples from two people who had been declared legally dead before receiving a pig heart and found that at the cellular level, rejection of xenotransplanted organs looks “very different” from that of organs transplanted from a human donor, Montgomery says. He adds that these findings could help researchers to anticipate rejection and develop tailored immunosuppressant regimens for future surgery. Vijy 1
BacktoCricaddict Posted July 2, 2024 Author Posted July 2, 2024 This is the absolute brilliance of science and technology at work to make human lives better. https://spectrum.ieee.org/prosthetic-leg Quote The Best Bionic Leg Yet. A surgical procedure and muscle-sensing electrodes allow neural control of a prosthetic limb For the first time, a small group of patients with amputations below the knee were able to control the movements of their prosthetic legs through neural signals—rather than relying on programmed cycles for all or part of a motion—and resume walking with a natural gait. The achievement required a specialized amputation surgery combined with a non-invasive surface electrode connection to a robotic prosthetic lower leg. A study describing the technologies was published today in the journal Nature Medicine. “What happens then is quite miraculous. The patients that have this neural interface are able to walk at normal speeds; and up and down steps and slopes; and maneuver obstacles really without thinking about it. It’s natural. It’s involuntary,” said co-author Hugh Herr, who develops bionic prosthetics at the MIT Media Lab.“Even though their limb is made of titanium and silicone—all these various electromechanical components—the limb feels natural and it moves naturally, even without conscious thought.” The approach relies on surgery at the amputation site to create what the researchers call an agonist-antagonist myoneural Interface, or AMI. The procedure involves connecting pairs of muscles (in the case of below-the-knee amputation, two pairs), as well as the introduction of proprietary synthetic elements. Prakat and ravishingravi 1 1
BacktoCricaddict Posted July 16, 2024 Author Posted July 16, 2024 https://nymag.com/intelligencer/article/cancer-treatment-immunotherapy-oncology-tcells-brain-tumor.html Cancer immunotherapy keeps delivering good news!! Prakat 1
BacktoCricaddict Posted July 17, 2024 Author Posted July 17, 2024 Another success in the fight against one of the most deadly infectious diseases - malaria (608,000 deaths per year). To me, it is also heartening to see the contribution of Indian science in this effort - the Serum Instt of India is manufacturing this vaccine. ------------------------------------------------------------------------------------------------------- https://www.unmc.edu/healthsecurity/transmission/2024/07/16/second-malaria-vaccine-launched-in-ivory-coast-marks-new-milestone/ The world’s second vaccine against malaria was launched on Monday as Ivory Coast began a routine vaccine program using shots developed by the University of Oxford and the Serum Institute of India. The introduction of the World Health Organization (WHO)-approved R21 vaccine comes six months after the first malaria vaccine, called RTS,S and developed by British drugmaker GSK, began being administered in a routine program in Cameroon. Some 15 African countries plan to introduce one of the two malaria vaccines this year with support from the Gavi global vaccine alliance. Ivory Coast has re
BacktoCricaddict Posted July 17, 2024 Author Posted July 17, 2024 @diga https://www.acsh.org/news/2024/07/12/aids-therapies-already-amazing-become-even-more-so-48866 The PURPOSE 1 trial, a Phase 3, double-blind, randomized study that began in 2021, evaluated the efficacy of lenacapavir – a capsid inhibitor – given by subcutaneous injection twice per year (!) against two commonly used PrEP drugs, Truvada and Descovy, both nucleoside reverse-transcriptase inhibitors (2). The results were astounding: 5,300 female study participants, aged 16-25, across 25 sites in South Africa and Uganda 2,134 received lenacapavir (two injections per year) 1,068 received Truvada (one pill per day) 2,136 received Descovy (one pill per day) 16 of the 1,068 women (1.5%) of the women who took Truvada became infected 39 of the 2,136 women who received Descovy (1.8%) became infected There were ZERO infections in the 2,134 women who received the lenacapavir injections If someone projected this back in the early 1990s when people were dying miserable (and inevitable) deaths, I would have suggested a psych evaluation. (When preliminary results became available, the trial was stopped early, and all the women switched to lenacapavir.) diga 1
diga Posted July 23, 2024 Posted July 23, 2024 Parkinsons disease & Marijuana... @BacktoCricaddict ... I understand this may be temporary, but is there active research on this ? Man with Parkinson's tries marijuana for the first time pic.twitter.com/SzIgUKazLf — Crazy Clips (@crazyclips_) July 22, 2024
BacktoCricaddict Posted July 23, 2024 Author Posted July 23, 2024 1 hour ago, diga said: Parkinsons disease & Marijuana... @BacktoCricaddict ... I understand this may be temporary, but is there active research on this ? Man with Parkinson's tries marijuana for the first time pic.twitter.com/SzIgUKazLf — Crazy Clips (@crazyclips_) July 22, 2024 Yes, there is a good bit of research on PD with cannabis. The quick summary is that - it may be helpful with some of the symptoms of PD - like minor tremors, sleeplessness etc., - but the evidence to alleviate serious motor symptoms is just not there. To me the biggest issue with these remedies - even if there was an effect - is the lack of systematic study on dosages and side-effects. There are so many compounds within the plant (plants are chemical factories), the dosage of each will vary with varieties and growing methods, and we don't really know which specific chemicals are giving the effect. Plus it is impossible to do double-blind studies because - what are you going to give the control group - a joint made of palak soppu? This is why it is important to identify the active ingredient, purify it, formulate it and do double-blind clinical studies. Unfortunately, there are politicians and influencers who think rigorous clinical trials are a 3-letter agency conspiracy to keep remedies out of patients' hands, unless it is a Covid vaccine, in which case they think the 3-letter agency did not study it enough ;-).
BacktoCricaddict Posted September 27, 2024 Author Posted September 27, 2024 Stem cells reverse woman’s (Type 1) diabetes — a world first. She is the first person with type 1 diabetes to receive this kind of transplant. https://www.nature.com/articles/d41586-024-03129-3 Quote A 25-year-old woman with type 1 diabetes started producing her own insulin less than three months after receiving a transplant of reprogrammed stem cells1. She is the first person with the disease to be treated using cells that were extracted from her own body. “I can eat sugar now,” said the woman, who lives in Tianjing, on a call with Nature. It has been more than a year since the transplant, and, she says, “I enjoy eating everything — especially hotpot.” The woman asked to remain anonymous to protect her privacy. James Shapiro, a transplant surgeon and researcher at the University of Alberta in Edmonton, Canada, says the results of the surgery are stunning. “They’ve completely reversed diabetes in the patient, who was requiring substantial amounts of insulin beforehand.” The study, published in Cell today, follows results from a separate group in Shanghai, China, who reported in April that they had successfully transplanted insulin-producing islets into the liver of a 59-year-old man with type 2 diabetes2. The islets were also derived from reprogrammed stem cells taken from the man’s own body and he has since stopped taking insulin. The studies are among a handful of pioneering trials using stem cells to treat diabetes, which affects close to half a billion people worldwide. Most of them have type 2 diabetes, in which the body doesn’t produce enough insulin or its ability to use the hormone diminishes. In type 1 diabetes, the immune system attacks islet cells in the pancreas. Islet transplants can treat the disease, but there aren’t enough donors to meet the growing demand, and recipients must use immune-suppressing drugs to prevent the body from rejecting the donor tissue. Stem cells can be used to grow any tissue in the body and can be cultured indefinitely in the laboratory, which means they potentially offer a limitless source of pancreatic tissue. By using tissue made from a person’s own cells, researchers also hope to avoid the need for immunosuppressants. This is a huge deal, friends. Pancreatic-cell transplants are not easy. Please keep in mind - this is for Type 1 diabetes - in which the person's own immune system attacks pancreatic cells that produce insulin. No amount of diet/lifestyle will cure that. I know several people, including 2 grand-aunts who had this, and it was devastating. Tillu and coffee_rules 2
randomGuy Posted September 28, 2024 Posted September 28, 2024 (edited) On 9/27/2024 at 4:46 PM, BacktoCricaddict said: Stem cells reverse woman’s (Type 1) diabetes — a world first. She is the first person with type 1 diabetes to receive this kind of transplant. https://www.nature.com/articles/d41586-024-03129-3 This is a huge deal, friends. Pancreatic-cell transplants are not easy. Please keep in mind - this is for Type 1 diabetes - in which the person's own immune system attacks pancreatic cells that produce insulin. No amount of diet/lifestyle will cure that. I know several people, including 2 grand-aunts who had this, and it was devastating. This is age at death distribution chart of USA in 1950 n in 2015. 1. Life expectancy increased 2. The curve became steeper Mode(peak of the curve) shifted from 77 to 87. Meaning maximum people died at this age. In 2015, one may observe that mode is 87 and almost everyone died by age 100. One can guess where this is going. Modal value in next 60-70 years may shift to say 97, and almost everyone will die by age 105. I was reading that max life expectancy of a population is restricted at 100 yrs and max life of an individual restricted at 120-125 yrs. Although there is no consensus. Edited October 17, 2024 by randomGuy
BacktoCricaddict Posted October 17, 2024 Author Posted October 17, 2024 This is still in the early research phase and has a long way to go before it hits the clinic (rigorous animal trials, safety trials, efficacy trials must be done), but is very exciting from a scientific perspective. Especially for Type 1 Diabetes patients, this could be a boon! Quote https://www.nature.com/articles/d41586-024-03357-7 Scientists have designed a new form of insulin that can automatically switch itself on and off depending on glucose levels in the blood. In animals, this ‘smart’ insulin1 reduced high blood-sugar concentrations effectively while preventing levels from dropping too low. For people with diabetes, controlling blood-sugar levels is a crucial — but demanding — task. Insulin keeps blood glucose in check, helping to prevent the many long-term complications associated with high blood sugar, such as cardiovascular disease, chronic kidney disease, stroke and blindness. A large proportion of the estimated 422 million people with diabetes worldwide require insulin injections. But excess insulin can cause blood-sugar levels to dip too low, a condition called hypoglycaemia, putting people at risk of serious complications, such as loss of consciousness, seizures and even death. Even mild or moderate hypoglycaemia can cause anxiety, weakness and confusion. People with diabetes — particularly those with type 1 diabetes, who always need to inject insulin — can have drops in blood-glucose concentrations several times a week, says Michael Weiss, a biochemist and physician at Indiana University in Indianapolis. “It really impairs quality of life.” For decades, researchers have been working to develop a system that can automatically adjust insulin activity based on the amount of glucose in a person’s blood. One common approach has been to make a compound containing deposits that release insulin when glucose concentrations rise. But a key disadvantage of this method is its irreversibility — once insulin is released, it can’t be reined in. A sugar-sensitive switch The latest study, published today in Nature, gets around this issue by modifying insulin itself using glucose-sensitive components. Rita Slaaby, a principal scientist at pharmaceutical company Novo Nordisk in Bagsværd, Denmark, and her colleagues engineered an insulin molecule with a switch that turns its activity on and off in response to glucose levels in the blood.
BacktoCricaddict Posted November 15, 2024 Author Posted November 15, 2024 https://www.genengnews.com/topics/cancer/car-t-cell-therapy-shows-efficacy-in-young-patients-with-incurable-brain-cancer/ Quote Clinical trial results are showing the first successes against solid tumors for CAR-T cells. The findings offer hope for children with a group of deadly brain and spinal cord tumors, including a cancer called diffuse intrinsic pontine glioma, or DIPG. The immune-cell therapy shrank children’s brain tumors, restored neurologic function and—for one participant—erased all detectable traces of a brain cancer typically considered incurable. The findings are published in Nature in the paper, “Intravenous and intracranial GD2-CAR T cells for H3K27M+ diffuse midline gliomas.” Of the 11 participants who received CAR-T cells in the trial, nine showed benefits and had functional improvement in the disabilities caused by their disease. Four participants had the volume of their tumors reduced by more than half and one of those four participants had a complete response (meaning his tumor disappeared from brain scans). Although it is too soon to say whether he is cured, he is healthy four years after diagnosis. “This is a universally lethal disease for which we’ve found a therapy that can cause meaningful tumor regressions and clinical improvements,” said Michelle Monje, MD, PhD, professor of neurology at Stanford Medicine. “While there is still a long way to go to figure out how to optimize this for every patient, it’s very exciting that one patient had a complete response. I’m hopeful he has been cured.” The patient, 20-year-old Drew, wants his outcome to be the first of many. “I’m hoping they’ll learn from all my successes to help other kids,” he said. Prakat 1
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