Gene therapy corrects sickle cell disease in laboratory study
... Jude Children's Research Hospital have alleviated sickle
cell disease pathology. In their studies, the ... become an important treatment for the disease.
cell disease, which affects millions of people ... same genetic defect and symptoms as humans with sickle
cell disease. The scientists introduced the gene ...
Children's national co-leads nationwide study of landmark sickle cell treatment
... and umbilical cord blood transplants for severe sickle
cell disease . Children's National will join more ... trial of this treatment. If successful, the sickle
Cell Unrelated Transplant (SCURT) trial has the ... option to more severely affected patients. sickle
cell disease affects approximately 70,000 ...
Gaining ground on sickle cell disease
... Although sickle
cell disease is a single-gene disorder, its ... that could potentially be helpful in predicting sickle
cell disease severity, perhaps even leading to ... which are known to affect symptom severity in sickle
cell diseasewith some patients experiencing ...
Clumps of red and white blood cells may contribute to sickle cell disease
... HILL Its long been known that patients with sickle
cell disease have malformed, sickle-shaped red ... School of Medicine have shown that blood from sickle
cell patients also contains clumps, or ... blood cells have been found in whole blood from sickle
cell patients. The study also shows how the red ...
Ovarian transplantation: First baby is born after a new technique
... mother who had been menopausal for two years as a result of treatment for sickle
cell anaemia. After transplanting her own ovarian tissue she started ... had their ovaries frozen. In addition to the first patient, treated for sickle
cell anaemia, the second patient had been treated for periarteritis ...
Statistical tool could explain gene study variations
... by Dr. Abdullah Kutlar, hematologist/oncologist and director of the MCG sickle
Cell Center, aims to understand the genetics of stroke risk in children with sickle
cell disease. With funding from the National Institutes of Health, Dr. Xu ...
Diabetes makes it hard for blood vessels to relax
... also is associated with vascular problems related to aging, hypertension, sickle
cell disease, atherosclerosis and erectile dysfunction, Dr. Romero says. L-citrulline already is taken by some sickle
cell patients to reduce breath-taking fibrosis in their lungs. In addition ...
Children's Hospital Oakland scientists first to discover new source for harvesting stem cells
... likely that placental stem cells, like umbilical cord blood and bone marrow stem cells, can be used to cure chronic blood-related disorders such as sickle
cell disease, thalassemia, and leukemia.
The study, led by Children's Hospital & Research Center Oakland scientists Frans Kuypers, PhD, and ...
Simulated gene therapy
... out the bad for the good. If doctors could safely do this, they could treat or even cure diseases like cystic fibrosis, certain types of cancer, sickle
cell anemia, and a number of rare genetic disorders.
Safety is a primary concern when working with gene therapy. Some of the first attempts at ...
Empire State Stem Cell Board awards $12.7 million to Albert Einstein College of Medicine
... of Medicine of Yeshiva University funding for stem cell research. The new grants, totaling $12.7 million, will help create technologies for treating sickle
cell anemia, cancer, heart and liver disease, as well as obesity and hepatitis.
In applauding President Obama's executive order restoring federal ...
Fountain of Youth to be found in the anthill?
... aspect of how epigenetic factors can influence outcomes in behavior, morphology, and longevity.
Harpegnathos saltator (literally meaning "jumping sickle
jaw") is a primitive species of ant where workers are able to perform either reproductive or helper tasks. A worker can become a reproductive ...
LSUHSC awarded patent for compound inhibiting cancer and other diseases
... disease, diabetic retinopathy, macular degeneration, obesity, corneal neovascularization, benign tumors, hemangioma, arterial/venous malformations, sickle
cell anemia, sarcoidosis, syphilis, pseudoxanthoma elasticum, Pagets disease, vein occlusion in the eye, infections of the retina, ...
Consortium develops new method enabling routine targeted gene modification
... Joung. "OPEN should also stimulate additional research into the potential application of ZFNs for gene therapy of single-gene disorders, such as sickle
cell anemia and cystic fibrosis." Joung's lab has already begun to explore ways to further simplify the OPEN method so that it can be performed more ...
Consortium develops new method to manipulate genetic material
... Joung said. "OPEN should also stimulate additional research into the potential application of ZFNs for gene therapy of single-gene disorders, such as sickle
cell anemia and cystic fibrosis."
Scientists discover novel way to remove iron from ferritin
... medications that dramatically improve the removal of excess iron in patients diagnosed with blood diseases such as B-Thalassemia (Cooley's anemia) or sickle
The study appears in this month's issue of the Journal of Biological Chemistry and was conducted by Dr. Theil and her co-authors ...
AIBS to cohost symposium on evolution, disease and human health
... species. Romero-Steiner has also been involved in immunogenicity studies of people who have undergone bone marrow transplants and splenectomies, have sickle
cell anemia, or are HIV positive.
George Armelagos is the Goodrich C. White Professor of Anthropology and Department Chair at Emory ...
Seattle Children's Hospital leads $23.7 million NIH grant to study gene repair
... a wide range of diseases, Scharenberg believes single-gene inherited disorders of the lymph and bone-marrow systems such as immune deficiencies, sickle
Cell Disease and thalassemias are the best place to start. Collectively, these disorders are a major global disease burden in children. The target ...
New imaging technique tracks traffic patterns of white blood cells
... explored various mouse models including that of sickle
Cell disease which is a common inherited blood ... to the National Institutes of Health (NIH). sickle
Cell disease is a blood disorder that affects ... method, red blood cells in a mouse model of sickle
Cell disease were shown to interact specifically ...
Researchers show key protein necessary for normal development of red blood cells
... which play a role in genetic blood disorders like sickle
cell anemia and beta-thalassemia, have shown in ... to future gene therapies for patients with sickle
cell anemia and beta-thalassemia.
In the ... gene has clinical relevance to treatment of sickle
cell anemia and beta-thalassemia," said Joyce A. ...
Sickle cell disease corrected in human models using stem cell-based gene therapy
... and RNA interference to genetically reverse sickle
cell disease (SCD) in human cells. This research ... production of the abnormal hemoglobin that causes sickle
cell disease, a viral vector was introduced in ... hemoglobin and suppressed production of the sickle
shaped hemoglobin typical of the disease.
Sickle cell and protection against malaria
... against malaria given by carrying the gene for sickle
cell haemoglobin may involve the immune system. ... areas most afflicted by malaria the gene for sickle
cell hemoglobin (HbS) occurs very frequently. The ... (HbAS), i.e., have one normal and one sickle
gene, and previous work has suggested that there ...
Discovery Promises Simpler Therapy for Sickle Cell Disease
... new understanding of the causes for symptoms of sickle
cell disease, a condition affecting one in every ... National Academy of Sciences.
The symptoms of sickle
cell disease have generally been attributed to ... explain a long-standing puzzle: patients with sickle
cell exhibit extreme variability in the severity ...
Birth defects: 8 million annually worldwide
... disease that confers some protection against malaria -- specifically, sickle
cell, thalassemia, and glucose-6-phosphate dehydrogenase deficiency ... tube defects (323,904 births); the hemoglobin disorders thalassemia and sickle
cell disease (307,897 births); Down syndrome (217,293 births); and G6PD ...
Researchers pioneer new gene therapy technique using natural repair process
... will work in several other diseases, such as sickle
cell disease is a disorder in which blood cells ... disease.
In theory, blood stem cells with the sickle
cell mutation could be removed, treated with the ...
Circles Of DNA Might Help Predict Success Of Stem Cell Transplantation
... as part of therapy for a variety of diseases such as leukemia or sickle
cell disease. In these cases physicians eliminate the patients' own stem ... cancers (e.g., acute lymphoblastic leukemia) or red cell diseases (e.g., sickle
Because blood from the normal, healthy donors contained ...
Study finds mercury prevalent in many western fish
... level of mercury deemed "protective" for fish-eating mammals.
Conducting the study with Herlihy and Hughes were Spencer Peterson and John Van sickle
of the EPA National Health and Ecological Effects Research Laboratory, located on the OSU campus.
Mercury entering the water via the atmosphere ...
Jumping gene could provide non-viral alternative for gene therapy
... purposes as well as manipulating the immune response. However, it has broad applications for correcting single gene disorders, such as hemophilia, sickle
cell disease and muscular dystrophy. ...
UCLA paper examines how one disease may prevent another
... J.B. Haldane proposed that malaria was an evolutionary force for selecting malaria-resistant genes. He suggested that those carrying the gene for sickle
cell anemia were better able to survive in malaria-infected areas. Leprosy patients have severe immune defects and cutaneous anergy -- an inability ...
Scientists discover that widely available drug also helps fight kidney disease
... number of individuals affected by ADPKD is greater than the number affected by cystic fibrosis, muscular dystrophy, hemophilia, Down's syndrome, and sickle
cell anemia combined. The disease is characterized by the proliferation of cysts that eventually debilitate the kidney, causing kidney failure in ...
U-M scientists discover identifying markers for primitive blood-forming stem cells
... growth factors, it could help us learn how to manipulate and grow colonies of HSCs outside the body, which could lead to new medical treatments for sickle
cell anemia, leukemia and other types of cancer."
As with most discoveries in stem cell science, U-M scientists say their work raises new ...
Molecular machine may lead to new drugs to combat human diseases
... stages of the cycle have been crystallized previously, and the targeted trans-splicing technique has been used to repair hemoglobin infected with sickle
cell anemia. The new structure provides scientists with tools to expand on ways to harness this molecular machine, Golden said.
The other ...