//www.koonmotors.com/news/health_medicine/gene_therapy/ Read all about gene therapy including current medical research on switching therapeutic genes on and off, light-activated gene therapy and gene silencing. en-us Sun, 22 Oct 2023 12:18:15 EDT Sun, 22 Oct 2023 12:18:15 EDT 60 //www.koonmotors.com/images/scidaily-logo-rss.png//www.koonmotors.com/news/health_medicine/gene_therapy/ For more science news, visit ScienceDaily. //www.koonmotors.com/releases/2023/08/230830195934.htm A clinical trial has tested a potentially curative stem cell gene therapy for sickle cell disease. The results were promising. Wed, 30 Aug 2023 19:59:34 EDT //www.koonmotors.com/releases/2023/08/230830195934.htm //www.koonmotors.com/releases/2023/08/230811115507.htm Researchers have developed a new form of gene therapy that can stop cell division in fusion-driven cancer. Fri, 11 Aug 2023 11:55:07 EDT //www.koonmotors.com/releases/2023/08/230811115507.htm //www.koonmotors.com/releases/2023/08/230810141004.htm A change in just one letter in the code that makes up a cancer-causing gene can significantly affect how aggressive a tumor is or how well a patient with cancer responds to a particular therapy. A new, very precise gene-editing tool will enable scientists to study the impact of these specific genetic changes in preclinical models rather than being limited to more broadly targeted tactics, such as deleting the entire gene. Thu, 10 Aug 2023 14:10:04 EDT //www.koonmotors.com/releases/2023/08/230810141004.htm //www.koonmotors.com/releases/2023/08/230810110340.htm Researchers have made a remarkable step forward in finding a potential cure for a type of childhood kidney disease. Thu, 10 Aug 2023 11:03:40 EDT //www.koonmotors.com/releases/2023/08/230810110340.htm //www.koonmotors.com/releases/2023/08/230810110338.htm New research has successfully reversed hearing loss in mice. Scientists used a genetic approach to fix deafness in mice, restoring their hearing abilities in low and middle frequency ranges. Thu, 10 Aug 2023 11:03:38 EDT //www.koonmotors.com/releases/2023/08/230810110338.htm //www.koonmotors.com/releases/2023/08/230809164732.htm A study shows new steps toward more patients getting gene therapy. Wed, 09 Aug 2023 16:47:32 EDT //www.koonmotors.com/releases/2023/08/230809164732.htm //www.koonmotors.com/releases/2023/07/230727143923.htm In a step forward in the development of genetic medicines, researchers have developed a proof-of-concept model for delivering gene editing tools to treat blood disorders, allowing for the modification of diseased blood cells directly within the body. If translated into the clinic, this approach could expand access and reduce the cost of gene therapies for blood disorders, many of which currently require patients receive chemotherapy and a stem cell transplant. Thu, 27 Jul 2023 14:39:23 EDT //www.koonmotors.com/releases/2023/07/230727143923.htm //www.koonmotors.com/releases/2023/07/230712124752.htm Cancer therapies that target specific genetic abnormalities in tumors have revolutionized treatment possibilities over the past two decades. While quality of life and survival are improved with targeted therapies, relapse is common due to the evolution of new tumor cells that are resistant to the targeted therapy. A new study reveals how lung tumors may develop drug resistance over time, pointing to a protein, called APOBEC3A, that could be a promising target. Results may help researchers develop new solutions for tumor resistance to targeted cancer therapies. Wed, 12 Jul 2023 12:47:52 EDT //www.koonmotors.com/releases/2023/07/230712124752.htm //www.koonmotors.com/releases/2023/05/230530125435.htm A scientific team has designed a strategy to fight obesity and diabetes in mice through ex vivo gene therapy which consists of implanting cells that have been manipulated and transformed in order to treat a disease. This is the first study to apply the ex vivo gene therapy technique to generate and implant cells that express the CPT1AM protein, an enzyme that plays a decisive role in many metabolic diseases such as obesity. Tue, 30 May 2023 12:54:35 EDT //www.koonmotors.com/releases/2023/05/230530125435.htm //www.koonmotors.com/releases/2023/04/230404114220.htm Researchers uncover a mechanism in a retinal transporter protein that causes blindness, could lead to targeted therapies Tue, 04 Apr 2023 11:42:20 EDT //www.koonmotors.com/releases/2023/04/230404114220.htm //www.koonmotors.com/releases/2023/02/230217103942.htm Researchers used the drug fasudil to restore neurons and improve methamphetamine-induced cognitive dysfunction in a mouse model of schizophrenia. Fri, 17 Feb 2023 10:39:42 EST //www.koonmotors.com/releases/2023/02/230217103942.htm //www.koonmotors.com/releases/2023/01/230124101241.htm 研究人员发现了一种基因,是overexpressed in mutated IDH1. Studies in human cells and a novel mouse model both show that this gene, called ZMYND8, plays a critical role in the radiation resistance. When they knocked down the gene, the glioma cells became responsive to radiation treatment. Tue, 24 Jan 2023 10:12:41 EST //www.koonmotors.com/releases/2023/01/230124101241.htm //www.koonmotors.com/releases/2023/01/230111150800.htm Scientists have demonstrated in animal models the possibility of using lipid nanoparticles and messenger RNA, the technology underpinning COVID-19 vaccines, to treat blindness associated with a rare genetic condition. Wed, 11 Jan 2023 15:08:00 EST //www.koonmotors.com/releases/2023/01/230111150800.htm //www.koonmotors.com/releases/2022/12/221207141919.htm Research has found that misfolded proteins in liver cells contribute to the development of liver cancer, shedding new light on the mysterious origins of one of the world's deadliest diseases. The findings could also help improve the safety of certain gene therapies for hemophilia. Wed, 07 Dec 2022 14:19:19 EST //www.koonmotors.com/releases/2022/12/221207141919.htm //www.koonmotors.com/releases/2022/11/221109124356.htm Technology that can change skin tissue into blood vessels and nerve cells also shows promise as a treatment for traumatic muscle loss. Wed, 09 Nov 2022 12:43:56 EST //www.koonmotors.com/releases/2022/11/221109124356.htm //www.koonmotors.com/releases/2022/11/221103140754.htm A new treatment for neurological and psychiatric diseases, that works by reducing the excitability of overactive brain cells, has been developed. Thu, 03 Nov 2022 14:07:54 EDT //www.koonmotors.com/releases/2022/11/221103140754.htm //www.koonmotors.com/releases/2022/10/221026142748.htm A fault in cells that form a key part of the immune system can be repaired with a pioneering gene editing technique, finds new research demonstrated in human cells and mice. Wed, 26 Oct 2022 14:27:48 EDT //www.koonmotors.com/releases/2022/10/221026142748.htm //www.koonmotors.com/releases/2022/10/221020122945.htm A new study identifies common and rare gene mutations that impact radiation resistance and sensitivity, an important step toward providing more individualized and effective radiotherapy for patients with cancer. The discovery ultimately could allow radiation dosage to be calibrated based on genetic mutations. Radiotherapy continues to be delivered using generic schedules and doses, unlike newer targeted drug therapy that are guided by the genomics of an individual's cancer. Thu, 20 Oct 2022 12:29:45 EDT //www.koonmotors.com/releases/2022/10/221020122945.htm //www.koonmotors.com/releases/2022/10/221019172139.htm Research led by doctors and scientists have identified a gene that may provide a therapeutic target for the deadly, treatment-resistant brain cancer glioblastoma multiforme (GBM). Wed, 19 Oct 2022 17:21:39 EDT //www.koonmotors.com/releases/2022/10/221019172139.htm //www.koonmotors.com/releases/2022/10/221011105754.htm Adults with a genetic form of childhood-onset blindness experienced striking recoveries of night vision within days of receiving an experimental gene therapy. Tue, 11 Oct 2022 10:57:54 EDT //www.koonmotors.com/releases/2022/10/221011105754.htm //www.koonmotors.com/releases/2022/10/221010115330.htm The blood-brain barrier (BBB) is an imposing foe for gene therapy. Formed of cells wedged tightly together, the BBB keeps toxins and pathogens that may be present in the blood from entering brain tissue, but it also keeps out potential treatment for diseases that affect the central nervous system (CNS). Researchers have discovered some delivery vehicles -- known as adeno-associated viruses (AAV) -- that can cross the barrier under certain circumstances, but most of the time, AAVs are inefficient at ferrying gene therapies across. Investigators are now working to optimize AAVs as gene delivery vehicles, improving their efficiency and their potential to deliver drugs to treat brain cancers such as glioblastoma and genetic diseases that affect the central nervous system. Mon, 10 Oct 2022 11:53:30 EDT //www.koonmotors.com/releases/2022/10/221010115330.htm //www.koonmotors.com/releases/2022/09/220923153055.htm Researchers explain how precision genome editing agents have enabled precise gene correction and disease rescue in inherited retinal diseases (IRDs). Fri, 23 Sep 2022 15:30:55 EDT //www.koonmotors.com/releases/2022/09/220923153055.htm //www.koonmotors.com/releases/2022/08/220824103057.htm 研究人员已经开发出一种新的基因疗法可能从oach that shows promise for treating the dry form of Age Related Macular Degeneration (AMD) -- a progressive eye disease that affects up to 10% of adults over 65 years of age and is a leading cause of severe vision impairment and blindness in this age group. Wed, 24 Aug 2022 10:30:57 EDT //www.koonmotors.com/releases/2022/08/220824103057.htm //www.koonmotors.com/releases/2022/06/220606213449.htm A new study in mice finds that a gene therapy appears to correct a rare creatine deficiency disorder that commonly results in intellectual disabilities, problems with speech, involuntary movements and recurrent seizures. Mon, 06 Jun 2022 21:34:49 EDT //www.koonmotors.com/releases/2022/06/220606213449.htm //www.koonmotors.com/releases/2022/05/220510103009.htm Scientists reported on an experimental gene-therapy-like technique to restore the normal activity of the TCCF4 gene deficient in individuals with Pitt-Hopkins syndrome. Tue, 10 May 2022 10:30:09 EDT //www.koonmotors.com/releases/2022/05/220510103009.htm //www.koonmotors.com/releases/2022/04/220422094328.htm Pancreatic cancer has a significantly poor prognosis; therefore, the development of effective treatments is an unmet clinical need. The major drawback in this field was the lack of useful model animals, which delayed the establishment of markers for early diagnosis and therapeutic options. The research group established an effective carcinogenesis method with wild-type rats by selectively introducing oncogenes into the pancreas, using the pancreas-targeted hydrodynamic gene delivery method that has been developed by the group. Fri, 22 Apr 2022 09:43:28 EDT //www.koonmotors.com/releases/2022/04/220422094328.htm //www.koonmotors.com/releases/2022/04/220405084554.htm Researchers have revealed how an 'accordion effect' is critical to switching off genes, in a study that transforms the fundamentals of what we know about gene silencing. The finding expands our understanding of how we switch genes on and off to make the different cell types in our bodies, as we develop in the womb. Tue, 05 Apr 2022 08:45:54 EDT //www.koonmotors.com/releases/2022/04/220405084554.htm //www.koonmotors.com/releases/2022/04/220401094845.htm A novel angiogenic microRNA drug can be a new option for the treatment of ischemic cardiovascular disease, according to a new study. In the study, the researchers describe a novel nuclear acting microRNA. Fri, 01 Apr 2022 09:48:45 EDT //www.koonmotors.com/releases/2022/04/220401094845.htm //www.koonmotors.com/releases/2022/03/220329100013.htm The diagnosis is rare, but devastating -- children with congenital muscle disorders often never learn to walk. Until now, there was no chance of recovery, but researchers are now presenting a possible therapeutic approach. Tue, 29 Mar 2022 10:00:13 EDT //www.koonmotors.com/releases/2022/03/220329100013.htm //www.koonmotors.com/releases/2022/02/220228114422.htm 研究人员已经开发出一种技术,heteroduplex oligonucleotide (HDO), that silences certain genes whose high expression levels fuel disease. Adding a specific molecular tag allowed them to target the HDO to immune cells called lymphocytes safely and efficiently. Doing so with an HDO specific to a gene called Itga4 improved symptoms in a mouse model of multiple sclerosis, suggesting this technology may be developed to use in human immune disorders. Mon, 28 Feb 2022 11:44:22 EST //www.koonmotors.com/releases/2022/02/220228114422.htm //www.koonmotors.com/releases/2022/02/220216170214.htm Sickle cell anemia is an inherited blood disorder where red blood cells become sickle/crescent shaped. It causes frequent infections, swelling in the hands and legs, pain, severe tiredness and delayed growth or puberty. Treatment typically focuses on controlling symptoms and may include pain medicines during crises; hydroxyurea to reduce the number of pain episodes; antibiotics and vaccines to prevent bacterial infections and blood transfusions. While a remedy for this severe disease has remained elusive, a recent study, if proven applicable, may be a possible cure. Wed, 16 Feb 2022 17:02:14 EST //www.koonmotors.com/releases/2022/02/220216170214.htm //www.koonmotors.com/releases/2022/01/220124103920.htm Researchers have found that induced pluripotent stem cell--derived cardiomyocytes from a patient with arrhythmogenic cardiomyopathy recapitulate the reduced contractility and impaired desmosome assembly associated with this disease, providing a rapid and convenient platform for developing new treatments such as gene replacement therapy. Mon, 24 Jan 2022 10:39:20 EST //www.koonmotors.com/releases/2022/01/220124103920.htm //www.koonmotors.com/releases/2022/01/220119142840.htm Researchers found a previously unknown gene mutation that can cause an incurable heart condition called dilated cardiomyopathy. This gene, BAC5, is important for the movement of calcium ions in the heart muscle and calcium ions are what drives the pumping of the heart. The good news is that the investigators also found a way to fix the mutation through a novel gene therapy approach, demonstrating a potential treatment for this devastating disease. Wed, 19 Jan 2022 14:28:40 EST //www.koonmotors.com/releases/2022/01/220119142840.htm //www.koonmotors.com/releases/2022/01/220119090848.htm New research suggests how a newly developed gene therapy can treat Dravet syndrome, a severe form of epilepsy, and potentially prolong survival for people with the condition. Wed, 19 Jan 2022 09:08:48 EST //www.koonmotors.com/releases/2022/01/220119090848.htm //www.koonmotors.com/releases/2022/01/220104120616.htm Medical experts have developed a new pre-clinical gene therapy for a rare disorder, known as limb-girdle muscular dystrophy (LGMD) 2B, that addresses the primary cellular deficit associated with this disease. Using a single injection of a low dose gene therapy vector, researchers restored the ability of injured muscle fibers to repair in a way that reduced muscle degeneration and enhanced the functioning of the diseased muscle. The treatment was safe, attenuated fibro-fatty muscle degeneration, and restored myofiber size and muscle strength, according to a new study. Tue, 04 Jan 2022 12:06:16 EST //www.koonmotors.com/releases/2022/01/220104120616.htm //www.koonmotors.com/releases/2021/12/211213160126.htm An experimental gene therapy for sickle cell disease restored blood cells to their normal shape and eliminated severe pain crises for years after treatment, a multicenter study has found. Mon, 13 Dec 2021 16:01:26 EST //www.koonmotors.com/releases/2021/12/211213160126.htm //www.koonmotors.com/releases/2021/12/211213133342.htm Researchers have identified novel changes in gene network interactions associated with cancer that may lead to new treatment targets for chemotherapy. Their work shows that more than 90 percent of changes in gene network interactions in nine types of cancer studied are not detectable by current tests focused on changes in gene expression. Mon, 13 Dec 2021 13:33:42 EST //www.koonmotors.com/releases/2021/12/211213133342.htm //www.koonmotors.com/releases/2021/12/211208085956.htm Sonodynamic therapy uses ultrasound in combination with drugs to release harmful reactive oxygen species (ROS) at the site of a tumor. However, the treatment isn't very effective because cancer cells can activate antioxidant defense systems to counteract it. Now, researchers have breached these defenses with CRISPR/Cas9 gene editing, allowing sonodynamic therapy to effectively shrink tumors in a mouse model of liver cancer. Wed, 08 Dec 2021 08:59:56 EST //www.koonmotors.com/releases/2021/12/211208085956.htm //www.koonmotors.com/releases/2021/12/211202191146.htm The brain is indeed a target for treating ALS (amyotrophic lateral sclerosis), scientists have discovered. This flips a long-standing belief the disease starts in the spinal motor neurons and any therapy would need to target the spine as the key focus. The new study shows the brain degenerates early in ALS, sends warning signals and shows defects very early in the disease. Degeneration of brain motor neurons is not merely a byproduct of the spinal motor neuron degeneration, as had been previously thought. Thu, 02 Dec 2021 19:11:46 EST //www.koonmotors.com/releases/2021/12/211202191146.htm //www.koonmotors.com/releases/2021/10/211019082729.htm A novel computational platform identifies top-performing viral vectors that could deliver gene therapies to the retina with maximum efficiency and precision. Tue, 19 Oct 2021 08:27:29 EDT //www.koonmotors.com/releases/2021/10/211019082729.htm //www.koonmotors.com/releases/2021/10/211018105930.htm A recent study examining the therapeutic potential of photoreceptor precursors, derived from clinically compliant induced pluripotent stem cells (iPSC), has demonstrated the safety and therapeutic potential of clinically compliant iPSC-derived photoreceptor precursors as a cell replacement source for future clinical trials. Mon, 18 Oct 2021 10:59:30 EDT //www.koonmotors.com/releases/2021/10/211018105930.htm //www.koonmotors.com/releases/2021/10/211012095026.htm The immune system protects the body from cancer. To protect healthy body cells from its own immune system, they have developed a protective shield: the protein CD47 is a so called 'don't eat me' signal, which tells the immune cells to stand back. Tumor cells exploit this CD47-based protection strategy for evading the immune system, by increasing presentation of CD47 on their cell surface. A team has now developed a therapy concept for programming the tumor cells to produce on their own a CD47-blocking and immune-activation fusion protein. This therapy approach could stop tumor growth. Tue, 12 Oct 2021 09:50:26 EDT //www.koonmotors.com/releases/2021/10/211012095026.htm //www.koonmotors.com/releases/2021/08/210831131437.htm Researchers identify proteins that drive cancer stem cells. Targeting and suppressing a particular protein called galectin1 could provide a more effective treatment for glioblastoma, in combination with radiation therapy. Tue, 31 Aug 2021 13:14:37 EDT //www.koonmotors.com/releases/2021/08/210831131437.htm //www.koonmotors.com/releases/2021/08/210825143114.htm New research offers insight into how to treat endometriosis. The researchers performed genetic analyses of humans and rhesus macaques to identify a specific gene, NPSR1, that increases risk of suffering from endometriosis. The results reveal a potential new nonhormonal drug target that may lead to improved therapy. Wed, 25 Aug 2021 14:31:14 EDT //www.koonmotors.com/releases/2021/08/210825143114.htm //www.koonmotors.com/releases/2021/08/210824135328.htm A new study has shown that the tumor-inhibiting gene TET2 is silenced in a large fraction of cases of acute lymphoblastic leukemia (ALL) in children. The scientists show that the gene can be reactivated by treatment with an existing drug, 5-azacytidine. The results suggest that 5-azacytidine may function as targeted therapy for ALL in children. Tue, 24 Aug 2021 13:53:28 EDT //www.koonmotors.com/releases/2021/08/210824135328.htm //www.koonmotors.com/releases/2021/07/210728111307.htm In a major advancement in the field of gene therapy for rare and devastating diseases, researchers have developed a "dimmer switch" system that can control levels of proteins expressed from gene therapy vectors. The system is based on alternative RNA splicing using an orally available small molecule and works effectively in tissues throughout the body, including the brain. Wed, 28 Jul 2021 11:13:07 EDT //www.koonmotors.com/releases/2021/07/210728111307.htm //www.koonmotors.com/releases/2021/07/210712092230.htm A novel method of gene therapy is helping children born with a rare genetic disorder called AADC deficiency that causes severe physical and developmental disabilities. The study offers new hope to those living with incurable genetic and neurodegenerative diseases. Mon, 12 Jul 2021 09:22:30 EDT //www.koonmotors.com/releases/2021/07/210712092230.htm //www.koonmotors.com/releases/2021/07/210708143918.htm Light therapy can help improve the mood of people with seasonal affective disorder (SAD) during short winter days, but exactly how this therapy works is not well understood. A new study finds that light therapy's beneficial effects come from activating the circadian clock gene Period1 in a part of the brain involved in mood and sleep-wake cycles. 星期四,08年7月2021 14:39:18美国东部时间 //www.koonmotors.com/releases/2021/07/210708143918.htm //www.koonmotors.com/releases/2021/06/210630091350.htm Amyotrophic lateral sclerosis, or ALS, attacks nerve cells known as motor neurons in the brain and spinal cord, gradually leading to paralysis. The loss of function of an important gene, C9orf72, may affect communication between motor neurons and muscles in people with this disease. Wed, 30 Jun 2021 09:13:50 EDT //www.koonmotors.com/releases/2021/06/210630091350.htm //www.koonmotors.com/releases/2021/06/210609123409.htm Scientists have developed polypeptide-based materials that act as effective vectors for delivering gene therapies. The new platform enables the vectors to be adapted to suit the specific gene therapy cargo. Wed, 09 Jun 2021 12:34:09 EDT //www.koonmotors.com/releases/2021/06/210609123409.htm //www.koonmotors.com/releases/2021/06/210601135730.htm 研究人员已经开发了一个概念验证胎面tment for blood disorders like sickle cell disease and beta-thalassemia that could raise hemoglobin levels by activating production of both fetal and adult hemoglobin. Using a viral vector engineered to reactivate fetal hemoglobin production, suppress mutant hemoglobin, and supply functional adult hemoglobin, the researchers developed an approach that could produce more hemoglobin through a single vector. Tue, 01 Jun 2021 13:57:30 EDT //www.koonmotors.com/releases/2021/06/210601135730.htm //www.koonmotors.com/releases/2021/05/210519120726.htm Base editing is a novel gene editing approach that can precisely change individual building blocks in a DNA sequence. By installing such a point mutation in a specific gene, an international research team has succeeded in sustainably lowering high LDL cholesterol levels in the blood of mice and macaques. This opens up the possibility of curing patients with inherited metabolic liver diseases. Wed, 19 May 2021 12:07:26 EDT //www.koonmotors.com/releases/2021/05/210519120726.htm //www.koonmotors.com/releases/2021/05/210519080438.htm Researchers have found a key new piece of the puzzle in the quest to use gene therapy to enable people born deaf to hear. Wed, 19 May 2021 08:04:38 EDT //www.koonmotors.com/releases/2021/05/210519080438.htm //www.koonmotors.com/releases/2021/05/210518114219.htm An international team of scientists have developed an experimental direct-acting antiviral therapy to treat COVID-19. Traditional antivirals reduce symptoms and help people recover earlier. This next-generation antiviral approach used gene-silencing RNA technology called siRNA (small-interfering RNA) to attack the virus' genome directly, which stops the virus from replicating, as well as lipid nanoparticles to deliver the siRNA to the lungs, the critical site of infection. Tue, 18 May 2021 11:42:19 EDT //www.koonmotors.com/releases/2021/05/210518114219.htm //www.koonmotors.com/releases/2021/05/210511123731.htm An investigational gene therapy can safely restore the immune systems of infants and children who have a rare, life-threatening inherited immunodeficiency disorder, according to new research. The researchers found that 48 of 50 children who received the gene therapy retained their replenished immune system function two to three years later and did not require additional treatments for their condition, known as ADA-SCID. Tue, 11 May 2021 12:37:31 EDT //www.koonmotors.com/releases/2021/05/210511123731.htm //www.koonmotors.com/releases/2021/05/210511081158.htm Scientists have developed a novel genetic sensor called a 'CopyCatcher,' which capitalizes on CRISPR-based gene drive technology, to detect instances in which a genetic element is copied precisely from one chromosome to another throughout cells in the body of a fruit fly. Next-generation CopyCatcher systems have the potential to measure how often such perfect copying might take place in different cells of the human body. Tue, 11 May 2021 08:11:58 EDT //www.koonmotors.com/releases/2021/05/210511081158.htm //www.koonmotors.com/releases/2021/05/210510133140.htm Looking to safely block a gene linked to factors known to cause heart disease, scientists may have found a new tool - light. Mon, 10 May 2021 13:31:40 EDT //www.koonmotors.com/releases/2021/05/210510133140.htm //www.koonmotors.com/releases/2021/05/210504191620.htm Researchers have used gene therapy to prevent learning and memory loss in a mouse model of Alzheimer's disease, a key step toward eventually testing the approach in humans with the neurodegenerative disease. Tue, 04 May 2021 19:16:20 EDT //www.koonmotors.com/releases/2021/05/210504191620.htm //www.koonmotors.com/releases/2021/04/210428113710.htm Researchers have discovered an explanation for why cerebral cavernous malformations (CCMs) -- clusters of dilated blood vessels in the brain -- can suddenly grow to cause seizures or stroke. Specifically, they found that a specific, acquired mutation in a cancer-causing gene (PIK3CA) could exacerbate existing CCMs in the brain. Furthermore, repurposing an already existing anticancer drug showed promise in mouse models of CCMs in improving brain-vascular health and preventing bleeding into the brain tissue. Wed, 28 Apr 2021 11:37:10 EDT //www.koonmotors.com/releases/2021/04/210428113710.htm //www.koonmotors.com/releases/2021/04/210421124533.htm A new approach to gene editing using the CRISPR/Cas9 system bypasses disease-causing mutations in a gene, enabling treatment of genetic diseases linked to a single gene, such as cystic fibrosis, certain types of sickle cell anemia, and other rare diseases. The method involves inserting a new, fully functional copy of the gene that displaces the mutated gene. Wed, 21 Apr 2021 12:45:33 EDT //www.koonmotors.com/releases/2021/04/210421124533.htm