Research on treating disease by editing or delivering genes. Covers sickle-cell gene therapy, gene-editing nanoparticles, mRNA delivery, and blood-cell-inspired carriers.
The International HapMap Project aims to map the locations of representative tag SNPs in DNA samples from diverse populations. The project's results will increase the power and reduce the cost of future genetic association studies, significantly speeding up the discovery of genes involved in common diseases.
Researchers identified three strongly predictive genes - OPAL1, GNB2L1, and IL-10 receptor alpha - that were associated with better outcomes in pediatric ALL patients. These genes may help improve risk classification and outcome prediction for acute leukemia in children.
A study found that patients with high levels of low molecular weight cyclin E have aggressive, invasive breast cancer, and the protein may be a better predictor of patient outcome than current markers. This altered form of cyclin E can induce genetic instability, making tumors unresponsive to treatment.
A study published in Cancer Research found that combining gene therapy and radiation therapy showed significant declines in PSA levels and eliminated cancer in many patients. The novel treatment has shown promise in treating aggressive prostate cancer, but more research is needed before it can be widely adopted.
Researchers successfully transferred and expressed MGMT into relatively few hematopoietic stem cells using a lentivirus vector, enabling gene-corrected cells to repopulate the hematopoietic compartment. This breakthrough has significant implications for human clinical trials of gene therapy in bone marrow transplantation settings.
Researchers found that blocking laminin-8 gene expression significantly reduced glioma cells' ability to invade neighboring tissue. The study supports the hypothesis that laminin-8 is involved in tumor spread and suggests a potential target for intervention strategies.
A recent study found that patients with a genetic subtype of diabetes have a significantly greater response to sulphonylurea drugs compared to those without a defined cause. This discovery highlights the importance of individualizing treatment for patients with type 2 diabetes, depending on their underlying cause.
The University of Pittsburgh has been designated as a cooperative research center to develop treatments for muscle-wasting diseases. The center will focus on gene and stem cell therapies to treat Duchenne muscular dystrophy, the most common and debilitating childhood disease.
Researchers used alginate, a major constituent of brown algae, to block viral leaks and reduce the concentration of toxic protein IL-12 in mice's livers. The solution improved the effectiveness of gene therapy by preventing sudden animal deaths caused by uncontrolled protein expression.
Researchers at Project A.L.S. have successfully tested a gene therapy approach that delays disease progression and prolongs survival in a mouse model of ALS. The treatment uses an adeno-associated viral vector to express the IGF1 protein, promoting neuronal survival.
Researchers found that injecting a gene producing insulin-like growth factor-1 (IGF-1) into muscles delayed disease onset by 31 days, preserved nerve cells, and reduced muscle wasting. This study may lead to a new, gene-based treatment for ALS affecting over 30,000 Americans.
Researchers at Johns Hopkins and Salk Institute develop gene therapy that slows progression of amyotrophic lateral sclerosis (ALS), a devastating disease affecting thousands. In experiments with mice, injection of insulin-like growth factor-1 (IGF-1) into muscles extends survival and improves strength.
Researchers develop novel gene therapy platform using AAV to deliver galanin, a neuroactive peptide that suppresses seizure activity. The treatment reduces sensitivity to focal seizures in laboratory rats, offering hope for treating temporal lobe epilepsy patients.
David Botstein, known as 'The Father of Modern Genetics', was awarded the 2003 Gruber Prize for his groundbreaking work on human genetic mapping and the discovery of transposons in bacteria. He will continue to lead research at Princeton University's Lewis-Sigler Institute for Integrative Genomics.
Researchers developed a gene therapy technique that uses an oncoretrovirus to transfer MGMT into normal bone marrow cells, allowing for the enrichment of healthy cells. The treatment showed promising results in mice with beta-thalassemia, achieving successful in vivo selection in 66% of cases.
Scientists have identified a preference for insertion at the beginning of genes and near actively expressed genes, which may explain why gene therapy patients developed leukemia. The discovery could lead to improved gene therapy techniques that insert genes in less risky areas.
A study by Temple University researchers found that pRb2/p130 binds to the estrogen receptor alpha and recruits molecules that make breast tumors more responsive to treatment. However, mutated pRb2/p130 signals can silence the expression of the estrogen receptor, making therapies less effective against cancer cells.
Weizmann Institute researchers have solved the 3D structure of the glucocerebrosidase enzyme involved in Gaucher disease. The discovery may lead to the design of more effective therapies, including enzyme replacement therapy and small molecule supplements.
Scientists have successfully induced the growth of new sensory hair cells in adult guinea pigs using gene therapy. The Math1 gene was inserted into non-sensory epithelial cells lining the inner ear, leading to the formation of new hair cells and attracting the growth of new fibers from auditory neurons.
Researchers discovered mutations in 14 tyrosine kinase genes, which play key roles in controlling cell growth and differentiation. These findings open the door to individualized treatment approaches for colorectal cancer, allowing for personalized therapeutics tailored to each patient's tumor DNA.
Researchers at Brenner Children's Hospital have developed a new gene therapy technology that uses highly efficient protein delivery to correct mitochondrial defects. This approach has the potential to prevent or treat genetic diseases like SIDS and TFP deficiency in infants, as well as heart and brain diseases later in life.
Researchers successfully integrated genes into hematopoietic stem cells to produce normal red blood cells in mice with beta-thalassemia and sickle cell disease. The technique uses a non-pathogenic AIDS virus gene to ferry DNA into the cells, selectively enriching genetically modified stem cells.
Scientists create hybrid virus that can attach to lung cells' top surface, allowing healthy genes to enter and correct genetic defects associated with cystic fibrosis. The new approach increases production of viral particles, a crucial step towards preclinical studies.
A recent Phase I study of compacted DNA gene therapy for cystic fibrosis demonstrated safety and tolerability. The treatment resulted in a meaningful increase in chloride ion transport in the nose, suggesting successful gene transfer and potential therapeutic benefits.
Researchers have discovered that injecting herpes simplex virus vectors can deliver neurotrophic factors to damaged nerves, improving intracavernous pressure in rats. This concept could translate to humans, enabling gene therapy as a prophylactic treatment for men undergoing radical prostatectomy.
The BP1 gene is found to be active in 57% of Caucasian women and 89% of African American women with breast cancer. Gene therapy targeting BP1 may help treat non-hereditary breast cancer, which accounts for 95% of cases.
Researchers identified 124 genes whose changes in expression differed depending on the treatment regimen, providing new clues on how medications work and how cancer cells protect themselves. The study's findings will help design more rational and effective ways to use drugs for deadly childhood leukemia.
Researchers have developed a new method using titanium oxide nanocrystals to deliver genes into cells, potentially overcoming current limitations of gene therapy. The nanocomposites can separate when exposed to light or x-rays, allowing for targeted gene delivery.
A France-based research team has discovered the gene responsible for Hutchinson-Gilford Progeria, a disease that causes young victims to age five to ten times faster than normal. The discovery is a critical step toward developing therapies for the disorder and programs to screen individuals for the defective gene.
Researchers at Michigan State University have identified a gene responsible for Smith-Magenis Syndrome (SMS), a rare genetic disorder. The discovery suggests that primarily one gene contributes to the phenotype, rather than multiple genes as previously thought.
The Alliance has awarded grants to Dr. Andrew Davidoff, Dr. Thomas Griffith, and Dr. Jeffrey Bartlett to develop gene therapies targeting neuroblastoma, prostate cancer, and ovarian cancer. The research holds promise for improved quality of life and reduced side effects for cancer patients.
Researchers found that gene transfer by adenovirus is less efficient in aging rat heart cells, highlighting the need for new treatment strategies. Boosting integrin expression may help overcome this hurdle, enabling low-dose gene therapy to deliver beneficial genes.
A decade-long analysis of 500 families with Alzheimer's Disease suggests a future of early prediction and prevention. The findings lead to a new vision of medical care, applicable not only to Alzheimer's but also to other genetic disorders.
A team of scientists from the University of Wisconsin-Madison has developed methods for recombining DNA segments in human embryonic stem cells using homologous recombination. This technique allows for manipulating any part of the human genome to study gene function and mimic human diseases.
The discovery of neurofibromin regulation may help develop targeted therapies to block neurofibromin degradation in NF1 patients and treat cancers with amplified Ras activity. Nf1 heterozygous cells show increased sensitivity to growth factors, suggesting even diminished neurofibromin levels can affect normal cell behavior.
Purdue University researchers have discovered how RNA molecules bind energy-bearing ATP molecule, enabling physical work and potential applications in nanotechnology. The discovery sheds light on RNA's role in creation of living things and may unlock new methods for delivering therapeutic molecules.
Limited availability of genetic data hampers scientific progress in finding new therapeutic targets. The Lancet editorial emphasizes the need to share accumulated evidence and re-evaluate reporting standards.
USC researchers have successfully used gene therapy to prompt mouse cells to produce human collagen, a crucial step towards treating patients with dystrophic epidermolysis bullosa. This breakthrough could lead to the development of new treatments for this debilitating skin condition, which causes blisters and permanent scars.
Researchers at Vanderbilt University Medical Center have developed a novel gene therapy approach that repairs messenger RNA, which could lead to effective treatments for inherited diseases. The method uses ribozymes to correct defective genes and has shown promising results in animal models.
Researchers at Ohio State University successfully tested micro-sized gelatin particles that may deliver therapeutic genes to the glomerulus in the kidneys. The biodegradable particles dissolved quickly enough to pass through the glomerulus within 10 hours, suggesting they could be useful in transferring genes to tissues.
Researchers at Purdue University have simplified the outer shell of the Ebola virus, making it easier to produce in a lab and delivering genes to defective cells. This breakthrough could lead to treatments for diseases like cystic fibrosis and lung cancer.
A new gene therapy approach has been shown to virtually eliminate cancerous tumors in mice by boosting their immune systems. The treatment, which blocks a key immunosuppressor called TGF-beta, resulted in 70-80% survival rates in mice with melanoma and prostate cancer.
A new gene profiling technique could help predict which breast cancer patients will respond to hormonal therapies and which will require additional chemotherapy. The study aims to identify the cellular programs that must be engaged or shut off for aromatase inhibitors to be successful.
New research from University of Minnesota presents promising findings on unrelated donor transplants, including a study showing improved survival rates and quality of life for leukemia/lymphoma patients. Another study explores the use of bone marrow-derived stem cells to treat Parkinson's disease.
Researchers have found that blocking myostatin in mice with muscular dystrophy improves muscle function, providing a potential new treatment for the condition. However, further studies are needed to address concerns about the approach's limitations and potential side effects.
Researchers have developed a novel system of gene therapy that targets lung stem cells to treat cystic fibrosis. By using a natural compound found in the lungs, the therapy enables airway cells to take up the therapeutic gene and shows substantial recovery from the disease defect.
As new genetic components are identified, clinical services must adapt to cope with the increasing demand. The role of genetic information in managing inherited cancers is becoming increasingly important, enabling tailored therapies for individual patients.
Researchers have developed a gene therapy technique that integrates DNA without using viral vectors, inserting the DNA at known locations. The technique successfully integrated a gene coding for Factor IX into mouse DNA, making 12 times more protein than control mice.
Researchers successfully used gene therapy to change the brain's circuitry in rats with Parkinson's disease, producing nearly 70% fewer symptoms. The treatment involves altering glutamate and GABA signaling pathways to protect dopamine neurons from deterioration.
A new study using gene therapy has shown promise in treating Parkinson's disease by 'resetting' overactive brain cells. The therapy may arrest or delay disease progression, offering a safer and more powerful alternative to current treatments.
A team led by Purdue University's David Sanders has developed a promising system to deliver genes to diseased liver and brain cells using a modified Ross River Virus (RRV) as a Trojan horse. This approach avoids the harmful side effects associated with other retroviruses, bringing gene therapy closer to treating human diseases.
Researchers at the University of Florida have demonstrated a novel approach to overcoming gene therapy hurdles by attaching viruses to tiny manufactured balls coated with iron oxide. The microspheres can be targeted using magnets, allowing for more efficient delivery of corrective genes to specific organs or tissues.
Researchers develop novel approach to treat thalassemias by blocking aberrant RNA splicing. The new virus effectively repairs existing RNA, restoring correct hemoglobin production and offering a lifeline for patients.
Researchers have successfully delivered the full-length dystrophin gene to mice with muscular dystrophy using stripped-down vectors, restoring normal muscle function. The breakthrough could pave the way for human clinical trials to assess the safety of this method in patients.
A novel gene therapy technique developed by Stanford researchers allows for the integration of large genes into human chromosomes, enabling continuous production of proteins. This technique has the potential to treat a variety of diseases, including Duchenne's muscular dystrophy and cystic fibrosis.
Researchers have developed a gene therapy approach to treat mucopolysaccharidosis VII, a disorder affecting multiple organ systems, in dogs. The treatment involves four intravenous injections of a retroviral vector expressing canine beta-glucuronidase, resulting in normal enzyme activity and near-normal mobility in treated dogs.
Researchers developed a gene therapy combining radiation therapy and cisplatin to target tumor cells, resulting in significant regression with no additional toxicity. The treatment uses a modified cold virus to insert the TNF gene into tumor cells, enhancing anti-cancer effects.
The study discovered a novel gene, ADAM33, linked to asthma susceptibility and airway changes in asthmatic patients. This gene is involved in generating bronchial hyperresponsiveness and may underlie abnormalities in asthmatic airway function.
A study identified three gene expression patterns correlated with survival after chemotherapy, which outperformed the International Prognostic Index. The patterns revealed aspects of tumor biology affecting treatment response, including immune system involvement and tumor cell division.
Researchers have successfully used gene therapy to treat two young children with ADA-SCID, a rare form of SCID that requires regular injections of the bovine form of ADA enzyme. The new method involves removing bone marrow cells and engineering them to produce healthy immune cells, offering a potentially lower-risk alternative to bone ...