(Press-News.org) Potential gene therapy for Sickle cell disease
Sickle cell disease (SCD) is an autosomal recessive disorder caused by mutations in hemoglobin (HBB) that deform red blood cells. A small number of patients have been successfully treated with allogeneic hematopoietic stem cell (HSC) transplantation; however, there are several drawbacks and complications associated with this procedure, including graft vs. host disease and long-term immune suppression. Many of complications could potentially be avoided by the use of autologous HSC transplant, which uses patient cells that have been treated genetically modified to replace defective hemoglobin. In this issue of the Journal of Clinical Investigation , Zulema Romero and colleagues at UCLA investigated the utility of a genetic vector encoding a human hemoglobin gene engineered to impede sickle hemoglobin and prevent the sickling of red blood cells. The vector was used to efficiently transduce bone marrow cells from SCD patients and the transduced cells were successfully transplanted into immunocompromised mice, suggesting that this method could potentially be used to treat SCD.
TITLE: β-globin gene transfer in human bone marrow for sickle cell disease
AUTHOR CONTACT: Zulema Romero
University of California, Los Angeles (UCLA), Los Angeles, CA, USA
Phone: 1-310-794-1884; Fax: 310-267-2774; E-mail: zulemar@ucla.edu
View this article at: http://www.jci.org/articles/view/67930?key=31b284a91ef6d46fc863
Cross-species malaria immunity induced by chemically attenuated parasites
Malaria, a mosquito-born infectious disease, kills over 600,000 people every year. Research has focused on the development of a vaccine to prevent the disease; however, many malaria vaccines targeting parasite antigens have failed because the antigen targets are highly variable. Based on the observation that low-density infections can induce antibody-independent immunity to different malaria strains, Michael Good and colleagues at Griffith University in Australia created a vaccine using blood-stage malaria parasites that were attenuated with a chemical agent that keeps the parasite from multiplying. In this issue of the Journal of Clinical Investigation, they demonstrate that mice inoculated with a single species of attenuated parasite display immunity to multiple malaria species for over 100 days. These data indicate that vaccination with chemically attenuated parasites provides protective immunity and suggest that such vaccines could be used to target human malaria species.
TITLE: Cross-species malaria immunity induced by chemically attenuated parasites
AUTHOR CONTACT: Michael Batzloff
Griffith University, Gold Coast, AUS
Phone: 5552 9434; E-mail: m.batzloff@griffith.edu.au
View this article at: http://www.jci.org/articles/view/66634?key=c4d24ede72f2d54f5147
A potential gene therapy for Mucopolysaccharidosis Type IIIA
Mucopolysaccharidosis Type IIIA (MPSIIIA) is a metabolic disorder in which the body is missing an enzyme that is required to break down long chains of sugars known as glycosaminoglycans. Over time, the glycosaminoglycans collect in the body and cause damage, particularly in the brain. In this issue of the Journal of Clinical Investigation, Fàtima Bosch and colleagues at Universitat Autònoma de Barcelona in Spain developed a form of gene therapy to replace the enzyme that is missing in MPSIIIA. By injecting the replacement gene into the the cerebrospinal fluid that surrounds the brain and spinal cord, Bosch and colleagues found that they could successfully deliver a replacement gene to the brain in mice and dogs. This study demonstrates that gene therapy can be delivered to the brain through the cerebrospinal fluid and suggests that this approach could potentially be used as a therapy for MPSIIIA.
TITLE: Whole body correction of Mucopolysaccharidosis IIIA by intra-cerebrospinal fluid gene therapy
AUTHOR CONTACT: Fàtima Bosch
Universitat Autònoma de Barcelona, Bellaterra, UNK, ESP
Phone: 0034935814182; Fax: 0034935814180; E-mail: fatima.bosch@uab.es
View this article at: http://www.jci.org/articles/view/66778?key=d24b37bc3364f9761834
ALSO IN THIS ISSUE
TITLE: Developmental differences in IFN signaling affect GATA-1s induced megakaryocyte hyperproliferation
AUTHOR CONTACT: Alan Cantor
Children's Hospital Boston/Harvard Medical School, Boston, MA, USA
Phone: 617-919-2026; Fax: 617-730-0222; E-mail: alan.cantor@childrens.harvard.edu
View this article at: http://www.jci.org/articles/view/40609?key=bf056c76f25f2f38ba80
TITLE: Inactivation of specific beta-cell transcription factors in Type 2 diabetes
AUTHOR CONTACT: Roland Stein
Vanderbilt University Medical Center, Nashville, TN, USA
Phone: 615-322-7027; Fax: 615-322-7236; E-mail: roland.stein@vanderbilt.edu
View this article at: http://www.jci.org/articles/view/65390?key=10e9ec4fdc34c0b7fedb
TITLE: Colon cancer progression is driven by APEX1-mediated up-regulation of Jagged
AUTHOR CONTACT: Ho Jin You
Chosun University School of Medicine, Gwangju, KOR
Phone: 82-62-230-6337; Fax: 82-62-230-6337; E-mail: hjyou@chosun.ac.kr
View this article at: http://www.jci.org/articles/view/65521?key=e4dab4c22c7ba7ac357f
TITLE: Angiopoietin 2 mediates microvascular and hemodynamic alterations in sepsis
AUTHOR CONTACT: Christian Kupatt
Klinikum Grosshadern, Ludwig-Maximilians-University, Munich, DEU
Phone: +49-89-7095-6092; Fax: +49-89-7095-6075; E-mail: christian.kupatt@med.uni-muenchen.de
View this article at: http://www.jci.org/articles/view/66549?key=72b1105b45c139d4740b
TITLE: Neutrophil extracellular traps sequester circulating tumor cells and promote metastasis
AUTHOR CONTACT: Lorenzo Ferri
MUHC, Montreal, PQ, CAN
Phone: 514-934-1934 ext. 44327; Fax: 514-398-6769; E-mail: Lorenzo.ferri@mcgill.ca
View this article at: http://www.jci.org/articles/view/67484?key=c5d09b064fa708677328
TITLE: Lipocalin 2 deactivates macrophages and worsens pneumococcal pneumonia outcomes
AUTHOR CONTACT: Sylvia Knapp
Medical University Vienna, Vienna, AUT
Phone: +43-1-40400-5139; Fax: +43-1-40400-5167; E-mail: sylvia.knapp@meduniwien.ac.at
View this article at: http://www.jci.org/articles/view/67911?key=3b51a00fcbe441d92d74
TITLE: Gray platelet syndrome and defrective thrombo-inflammation in Nbeal2-deficient mice
AUTHOR CONTACT: Bernhard Nieswandt
University Hospital Würzburg and Rudolf Virchow Center, Würzburg, , DEU
Phone: +49 931 31 80405; Fax: +49 931 201 48123; E-mail: bernhard.nieswandt@virchow.uni-wuerzburg.de
View this article at: http://www.jci.org/articles/view/69210?key=0fc85302255cce6a5803
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JCI early table of contents for July 1, 2013
2013-07-01
ELSE PRESS RELEASES FROM THIS DATE:
Potential gene therapy for Sickle cell disease
2013-07-01
Sickle cell disease (SCD) is an autosomal recessive disorder caused by mutations in hemoglobin (HBB) that deform red blood cells. A small number of patients have been successfully treated with allogeneic hematopoietic stem cell (HSC) transplantation; however, there are several drawbacks and complications associated with this procedure, including graft vs. host disease and long-term immune suppression. Many of complications could potentially be avoided by the use of autologous HSC transplant, which uses patient cells that have been treated genetically modified to replace ...
Cross-species malaria immunity induced by chemically attenuated parasites
2013-07-01
Malaria, a mosquito-born infectious disease, kills over 600,000 people every year. Research has focused on the development of a vaccine to prevent the disease; however, many malaria vaccines targeting parasite antigens have failed because the antigen targets are highly variable. Based on the observation that low-density infections can induce antibody-independent immunity to different malaria strains, Michael Good and colleagues at Griffith University in Australia created a vaccine using blood-stage malaria parasites that were attenuated with a chemical agent that keeps ...
Senior moment? Stereotypes about aging can hurt older adults' memory, but there's an easy fix
2013-07-01
Of the many negative stereotypes that exist about older adults, the most common is that they are forgetful, senile and prone to so-called "senior moments." In fact, while cognitive processes do decline with age, simply reminding older adults about ageist ideas actually exacerbates their memory problems, reveals important new research from the USC Davis School of Gerontology.
The study, forthcoming in the journal Psychological Science, is an extension of the idea of "stereotype threat" — that when people are confronted with negative stereotypes about a group with which ...
How the body aids and abets the spread of cancer
2013-07-01
This news release is available in French.
Montreal, July 1st 2013 – The very system that is meant to protect the body from invasion may be a traitor. These new findings of a study, led by investigators at the Research Institute of the McGill University Health Centre (RI-MUHC), reveal that infection-fighting white blood cells play a role in activating cancer cells and facilitating their spread to secondary tumours. This research, published today in the Journal of Clinical Investigation has significant implications for both the diagnosis and treatment of cancer. ...
Climate change: Diseqilibrium will become the norm in the plant communities of the future
2013-07-01
The forest we are used to looking at is not at all in equilibrium. Since the Ice Age, a number of plants have been 'missing' in Northern Europe, i.e. species that have not yet arrived. The same applies in many other parts of the world. Similarly, there is evidence that -- even today -- it often takes a very long time before plants follow when glaciers retreat, or the climate changes. In future, such disequilibrium will become the norm in the plant communities on Earth. This has been demonstrated by a new synthesis carried out by two researchers at Aarhus University -- Professor ...
Children with delayed motor skills struggle more socially
2013-07-01
CORVALLIS, Ore. – Studies have shown that children with autism often struggle socially and now new research suggests that a corresponding lack of motor skills – including catching and throwing – may further contribute to that social awkwardness.
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Lead author Megan MacDonald is an assistant professor in the College of Public Health and Human Sciences at Oregon State University. She is an expert ...
UCLA discovery sheds light on why Alzheimer's meds rarely help
2013-07-01
BACKGROUND
The Alzheimer's Association projects that the number of people living with Alzheimer's disease will soar from 5 million to 13.8 million by 2050 unless scientists develop new ways to stop the disease. Current medications do not treat Alzheimer's or stop it from progressing; they only temporarily lessen symptoms, such as memory loss and confusion.
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DNA particles in the blood may help speed detection of coronary artery disease
2013-07-01
DALLAS, July 1, 2013 — DNA fragments in your blood may someday help doctors quickly learn if chest pain means you have narrowed heart arteries, according to a new study published in the American Heart Association journal Arteriosclerosis, Thrombosis, and Vascular Biology.
The study involved 282 patients, ages 34 to 83, who reported chest pain and were suspected of having coronary artery disease. Researchers used computed tomography imaging to look for hardened, or calcified, buildup in the blood vessels that supply the heart. Blood samples also were tested for bits of ...
New guidelines pave the road for achieving an AIDS-free generation
2013-07-01
KUALA LUMPUR—July 1, 2013—The Elizabeth Glaser Pediatric AIDS Foundation (EGPAF) welcomes the World Health Organization (WHO)'s new HIV/AIDS treatment and prevention guidelines. For the first time, the 2013 guidelines combine recommendations across the continuum of HIV care and prevention programs, including expanding treatment eligibility for HIV-positive pregnant women, mothers, and children. These recommendations signify a major step forward in the global effort to achieve an AIDS-free generation, but will require a significant shift in current implementation efforts. ...
Researchers have discovered a new proteasome regulatory mechanism
2013-07-01
Dysfunction of the ubiquitin-proteasome system is related to many severe neurodegenerative diseases, such as Alzheimer's and Parkinson's diseases, and certain types of cancer. Such dysfunction is also believed to be related to some degenerative muscle diseases.
The proteasome is a large protein complex that maintains cellular protein balance by degrading and destroying damaged or expired proteins. The ubiquitin is a small protein that labels proteins for destruction for the proteasome. If the system does not work effectively enough, expired and damaged proteins accumulate ...