Archive for the ‘Gene Therapy Research’ Category

Role of family history and genetics in carpal tunnel syndrome
Hand pain, gripping problems, wrist pain, tingling or numbness and waking up at night from any one of these sensations can be symptoms of carpal tunnel syndr...

By: CarpalRx

See original here:
Role of family history and genetics in carpal tunnel syndrome - Video

Genetics Video Pluripotent Stem Cells

By: Emerson Pk

Read the original:
Genetics Video Pluripotent Stem Cells - Video

Pete Hardy: A Patient #39;s Perspective on Adult Stem Cell Therapy
Pete Hardy, a passionate MedRebel and a 61-year-old patient advocate, voices his experience with adult stem cells. Watch his story! For more information see ...

By: Med Rebels

See original here:
Pete Hardy: A Patient's Perspective on Adult Stem Cell Therapy - Video

Ben Hoover and his news report on Eliza O #39;Neill and her battle with San Filippo Syndrome
Eliza has a play date in her Columbia backyard with friends at different stages of San Filippo Syndrome. I started reporting on Eliza and her family #39;s effort...

By: Ben Hoover

More:
Ben Hoover and his news report on Eliza O'Neill and her battle with San Filippo Syndrome - Video

Genetics History
I use this PowerPoint in my biology class at Beverly Hills High School. Topics: - Gregor Mendel - Genotype - Phenotype - Homozygous - Heterozygous - Punnett ...

By: Kyle Kobe

Continue reading here:
Genetics History - Video

Voki by Katarina - Genetics

By: Sam Moore

See the rest here:
Voki by Katarina - Genetics - Video

Paw Print Genetics partners with the Spokane Police Department #39;s K9 unit
Paw Print Genetics is partnering with the Spokane Police Department to improve the genetic health of dogs in the SPD K-9 Unit. Paw Print Genetics, a canine g...

By: Paw Print Genetics

Excerpt from:
Paw Print Genetics partners with the Spokane Police Department's K9 unit - Video

Exploring long-term expression of therapeutic transgenes
Results from early-phase clinical trials have indicated that recombinant adeno-associated viruses (rAAV) could potentially be used for gene therapy. In each ...

By: Journal of Clinical Investigation

Link:
Exploring long-term expression of therapeutic transgenes - Video

Gene therapies, today: Marina Cavazzana at TEDxLakeComo
Pediatrician and researcher. Director of the Department of Biotherapies at Hpital Necker in Paris, Marina Cavazzana has developed innovative biotherapies to...

By: TEDxTalks

Continue reading here:
Gene therapies, today: Marina Cavazzana at TEDxLakeComo - Video

Gene Therapy Speech: COM 101
by Gabriella de Souza.

By: Gabby de Souza

Read the original here:
Gene Therapy Speech: COM 101 - Video

Human Treg Responses Allow Sustained Recombinant Adeno-Associated VirusMediated Transgene Expression Recombinant adeno-associated virus (rAAV) vectors have shown promise for the treatment of several diseases; however, immune-mediated elimination of transduced cells has been suggested to limit and account for a loss of efficacy. To determine whether rAAV vector expression can persist long term, researchers administered rAAV vectors expressing normal, M-type ?-1 antitrypsin to AAT-deficient subjects at various doses by multiple i.m. injections. [J Clin Invest] Abstract | Press Release

SUMO-1 Gene Transfer Improves Cardiac Function in a Large-Animal Model of Heart Failure Toward clinical translation, scientists evaluated the effects of small ubiquitin-related modifier 1 (SUMO-1) gene transfer in a swine model of ischemic heart failure. After gene delivery, there was a significant increase in the maximum rate of pressure rise that was most pronounced in the group that received both SUMO-1 and SERCA2a. [Sci Transl Med] Abstract | Press Release

Aldehyde Dehydrogenase Expression Drives Human Regulatory T Cell Resistance to Posttransplantation Cyclophosphamide Scientists studied early T cell reconstitution in patients undergoing allogeneic blood or marrow transplantation with posttransplantation cyclophosphamide and the effects of mafosfamide, a cyclophosphamide analog, on CD4+ T cells in allogeneic mixed lymphocyte reactions in vitro. [Sci Transl Med] Abstract | Press Release

Hypoxic Mesenchymal Stem Cells Engraft and Ameliorate Limb Ischemia in Allogeneic Recipients Investigators evaluated the therapeutic potential of mesenchymal stem cells cultured under a different environment in ameliorating limb ischemia in allogeneic recipients. [Cardiovasc Res] Abstract

Guidance of In Vitro Migration of Human Mesenchymal Stem Cells and In Vivo Guided Bone Regeneration Using Aligned Electrospun Fibers Scientists tested their hypothesis that cell migration and bone tissue formation can be guided and facilitated by microscale morphological cues presented from a scaffold. [Tissue Eng Part A] Abstract

Allogeneic Human Mesenchymal Stem Cell Therapy (Remestemcel-L, Prochymal) as a Rescue Agent for Severe Refractory Acute GvHD in Pediatric Patients Investigators evaluated the risk/benefit profile of remestemcel-L (Prochymal), a third party, off-the-shelf source of human mesenchymal stem cells, as a rescue agent for treatment-resistant acute graft versus host disease (GvHD) in pediatric patients. [Biol Blood Marrow Transplant] Abstract | Press Release

MSCs vs. MSCs Combined with CB for Engraftment Failure Following Autologous Hematopoietic Stem Cell Transplantation: A Pilot Prospective, Open-Labeled, Randomized Trial Researchers prospectively evaluated the effects and safeties of mesenchymal stem cells (MSCs) alone and combined with cord blood (CB) for engraftment failure. Twenty two patients were randomized to receive MSCs or MSCs plus CB. If patients did not response to MSCs, they would receive the therapeutic schedule in CB group, and those patients with partial response in MSCs group and those without complete response in CB group would continue another cycle of MSCs treatment. [Biol Blood Marrow Transplant] Abstract

Extrahepatic Stem Cells Mobilized from the Bone Marrow by the Supplementation of BCAA Ameliorate Liver Regeneration in an Animal Model Researchers report that liver repopulation could be achieved with hepatocytes that bone marrow derived from stem cells proliferated. The treatment by oral supplementation of beanched-chain amino acids (BCAA) resulted in higher levels of CD34+SDF+c-kit+ stem cells in the blood and liver after liver transplantation. [J Gastroenterol Hepatol] Abstract

A Novel Three-Dimensional Adipose-Derived Stem Cell Cluster for Vascular Regeneration in Ischemic TissueThe authors describe an innovative three-dimensional cell mass (3DCM) culture that is based on cell adhesion (basic fibroblast growth factor-immobilized substrate) and assess the therapeutic potential of 3DCMs composed of human adipose tissue-derived stromal cells. [Cytotherapy] Abstract

Human Salivary Gland Stem Cells Ameliorate Hyposalivation of Radiation-Damaged Rat Salivary Glands Researchers isolated tissue-specific stem cells from the human submandibular salivary gland (hSGSCs). Transplantation of hSGSCs to radiation-damaged rat salivary glands rescued hyposalivation and body weight loss, restored acinar and duct cell structure, and decreased the amount of apoptotic cells. [Exp Mol Med] Abstract | Full Article

Continued here:
CellTherapyNews — Cell Therapy News Home

Gene therapy is using "genes as medicine". It is an experimental approach to treating genetic disease where the faulty gene is fixed, replaced or supplemented with a healthy gene so that it can function normally. Most genetic diseases cannot be treated, but gene therapy research gives some hope to patients and their families as a possible cure. However, this technology does not come without risks and many clinical trials to evaluate its effectiveness need to be done before gene therapy can be put to regular medical use.

To get a new gene into a cell's genome, it must be carried in a molecule called a vector. The most common vectors currently being used are viruses, which naturally invade cells and insert their genetic material into that cell's genome. To use a virus as a vector, the virus' own genes are removed and replaced with the new gene destined for the cell. When the virus attacks the cell, it will insert the genetic material it carries. A successful transfer will result in the target cell now carrying the new gene that will correct the problem caused by the faulty gene.

Viruses that can be used as vectors include retroviruses like HIV, adenoviruses (one of which causes the common cold), adeno-associated viruses and herpes simplex viruses. There are also many non-viral vectors being tested for gene therapy uses. These include artificial lipid spheres called liposomes, DNA attached to a molecule that will bind to a receptor on the target cell, artificial chromosomes and naked DNA that is not attached to another molecule at all and can be directly inserted into the cell.

The actual transfer of the new gene into the target cell can happen in two ways: ex vivo and in vivo. The ex vivo approach involves transferring the new gene into cells that have been removed from the patient and grown in the laboratory. Once the transfer is complete, the cells are returned to the patient, where they will continue to grow and produce the new gene product. The in vivo approach delivers the vector directly to the patient, where transfer of the new gene will occur in the target cells within the body.

Conditions or disorders that result from mutations in a single gene are potentially the best candidates for gene therapy. However, the many challenges met by researchers working on gene therapy mean that its application is still limited while the procedure is being perfected.

Before gene therapy can be used to treat a certain genetic condition or disorder, certain requirements need to be met:

Clinical trials for gene therapy in other countries (for example France and the United Kingdom) have shown that there are still several major factors preventing gene therapy from becoming a routine way to treat genetic conditions and disorders. While the transfer of the new gene into the target cells has worked, it does not seem to have a long-lasting effect. This suggests that patients would have to be treated multiple times to control the condition or disorder. There is also always a risk of a severe immune response, since the immune cells are trained to attack any foreign molecule in the body. Working with viral vectors has proven to be challenging because they are difficult to control and the body immediately recognizes and attacks common viruses. Recent work has focussed on potential non-viral vectors to avoid the complications associated with the viral vectors. Finally, while there are thousands of single-gene disorders, the more common genetic disorders are actually caused by multiple genes, which do not make them good candidates for gene therapy.

One promising application of gene therapy is in treating type I diabetes. Researchers in the United States used an adenovirus as a vector to deliver the gene for hepatocyte growth factor (HGF) to pancreatic islet cells removed from rats. They injected the altered cells into diabetic rats and, within a day, the rats were controlling their blood glucose levels better than the control rats. This model mimics the transplantation of islet cells in humans and shows that the addition of the HGF gene greatly enhances the islet cells' function and survival.

In Canada, researchers in Edmonton, Alberta also developed a protocol to treat type I diabetes. Doctors use ultrasound to guide a small catheter through the upper abdomen and into the liver. Pancreatic islet cells are then injected through the catheter into the liver. In time, islets are established in the liver and begin releasing insulin.

Another application for gene therapy is in treating X-linked severe combined immunodeficiency (X-SCID), a disease where a baby lacks both T and B cells of the immune system and is vulnerable to infections. The current treatment is bone marrow transplant from a matched sibling, which is not always possible or effective in the long term. Researchers in France and the United Kingdom, knowing the disease was caused by a faulty gene on the X chromosome, treated 14 children by replacing the faulty gene ex vivo. Upon receiving the altered cells, the patients showed great improvements in their immune system functions. Unfortunately, two of the children developed a form of leukemia several years after the treatment. Further investigation showed that the vector had inserted the gene near a proto-oncogene, which led to uncontrolled growth of the T cells. The clinical trials were put on hold until a safer method can be designed and tested.

Here is the original post:
Gene Therapy - Biotechnology - Science and Research

The mission of the Center for Gene Therapy is to investigate and employ the use of gene and cell based therapeutics for prevention and treatment of human diseases including: neuromuscular and neurodegenerative diseases, lysosomal storage disorders, ischemia and re-perfusion injury, neonatal hypertension, cancer and infectious diseases.

Learn about our areas of focus and featured research.

The National Institutes of Health has designated the Center for Gene Therapy as a Paul D. Wellstone Muscular Dystrophy Cooperative Research Center (MDCRC). MDCRCs promote basic, translational and clinical research and provide important resources that can be shared within the national muscle biology and neuromuscular research communities.

The MDCRC will allow Nationwide Children's researchers to further develop methods to overcome immune barriers to gene correction for Duchenne muscular dystrophy.

The Center for Gene Therapy and the Viral Vector Core are home to a Good Manufacturing Practice (GMP) production facility for manufacture of clinical-grade rAAV vectors.

Investigators with the Center for Gene Therapy currently are conducting numerous clinical research studies, especially for neuromuscular disorders.

The OSU and Nationwide Children's Muscle Group brings together investigators with diverse research interests in skeletal muscle, cardiac muscle, and neuromuscular biology.

Learn how the 24 labs within OSU/Nationwide Children's Muscle Group are working to improve approaches to treat muscle injury and disease. Read about how their collaborations are changing the way we treat neuromuscular diseases.

Hosted by Kevin Flanigan, MD, "This Month in Muscular Dystrophy" podcasts highlight the latest in muscular dystrophy and other inherited neuromuscular disease research. During each podcast, authors of recent publications discuss how their work improves our understanding of inherited neuromuscular diseases, and what their work might mean for treatment of these diseases.

Originally posted here:
Center for Gene Therapy :: The Research Institute at ...

By its very nature, gene therapy of genetic diseases is a technology of tremendous potential in relieving human suffering, but it is at the same time a massive and dauntingly complex scientific endeavor. That we currently stand on the threshold of successfully curing genetic diseases is a monument to the unprecedented scientific breakthroughs achieved in recent years, in mapping the genetic mutations underlying inherited metabolic diseases and in precisely defining the pathophysiology of the resulting cellular defects. These accomplishments have required the concerted effort of scores of dedicated clinical and basis science researchers, many of whom are faculty members at the University of Iowa. However, despite the recent advances, the science for gene therapy of human genetic diseases is still currently in its infancy and much work remains to be done. The Iowa Center for Gene Therapy has a particular emphasis on cystic fibrosis, but extends as well to other areas in which the University of Iowa has particular strengths. Historically, the academic culture at The University of Iowa has been one of collaboration and the sharing of resources and expertise, and gene therapy research for CF has benefited from the active gene therapy programs directed at other target organs and diseases. It is the goal of this Center to further facilitate such interactions among researchers investigating gene therapy for diseases affecting the CNS, muscle, vessels, skin, liver, and lung.

Steps for Gene Therapy Research

There are three broad research areas underlying the development of successful gene therapy approaches for the treatment of inherited metabolic diseases:

(1) The first is the identification of disease-causing gene mutations.

(2) The pathophysiology arising from the genetic mutation must then be studied in the context of gene function and the basic cell biology of the affected organ system(s), so that the appropriate cellular targets for gene therapy can be identified.

(3) Lastly, suitable and effective gene therapy vector systems for targeting the specific affected system and providing long-term amelioration of the disease must be developed.

Cystic Fibrosis: The Disease

Cystic Fibrosis is the most common fatal genetic disease in the Caucasian population, with a frequency of about one in 2,500 live births a year. Currently, the median age of survival for a patient with CF is 31 years. Although disseminated throughout other organs, the most life-threatening clinical feature of CF is pulmonary obstruction caused by abnormally thick mucus secretions and chronic infection by opportunistic bacteria, such as Pseudomonas and Staphlococcus, which lead to respiratory failure. Treatment today consists of a comprehensive approach, including postural drainage and percussion, replacement of pancreatic enzymes and proper nutrition, administration of antibiotics, mucus-thinning and anti-inflammatory drugs, and newer drugs aimed at symptomatic correction. In 1989, the defective gene underlying CF was identified as Cystic Fibrosis Transmembrane Conductance Regulator (CFTR), opening a new era of investigation into the pathophysiological mechanisms of CF disease, and leading to the promise of gene transfer as the ultimate therapeutic intervention. CFTR is a cAMP-regulated Cl- channel and also has been implicated in the regulation of other ion channels localized in the apical membranes of airway epithelial cells. Mutations in CFTR result in defective ion transport, leading to thick mucus, impaired mucociliary clearance and decreased bacterial killing. Despite recent progress in associating CF defects with CFTR dysfunction, there remain many unanswered questions concerning the roles of CFTR in normal airway biology and CF pathology, and the identification of the relevant cellular targets for gene therapy.

The University of Iowa has a long-standing history as a leader in the field of CF research and applied gene therapies for this disorder. In this regard the University of Iowa has several funded CF research programs, including a Research Development Program (RDP) funded by the Cystic Fibrosis Foundation, a CF Scientific Center of Research (SCOR) funded by NHLBI, and a Gene Therapy for Cystic Fibrosis program project grant (PPG) funded by NIH, all under the direction of Michael Welsh. These programs are an integral part of the CF research base involved in the Iowa Center for Gene Therapy of Cystic Fibrosis. In three Clinical Trials for gene therapy of CF, Dr. Michael Welshs group and colleagues in the CF Clinical Center have evaluated the safety and efficacy of CFTR gene delivery to the nasal mucosa of CF patients with recombinant adenoviral vectors and cationic liposomes (1, 2, 3, 4, 5). Importantly, the CF research community at the University of Iowa has also made significant contributions to increase understanding of lung development and the identification of putative stem cell targets for gene therapy, as well as the recent identification of new hypotheses regarding the pathogenesis of bacterial infection in CF airways. This group of investigators has also been instrumental in developing animal and in vitro model systems critical for studying disease pathogenesis and for testing treatment strategies and surrogate endpoints. Ongoing research programs in vector development, vector-host interactions and virology have aided in understanding some of the present limitations in current vectors. Studies are now directed toward improving the efficacy of several vector systems (adenoviruses, adeno-associated viruses [AAV], retroviruses, and non-viral vectors) for gene transfer to multiple organs. Development of an effective gene therapy for cystic fibrosis (CF) requires further advancement in areas of basic research on airway biology, CF pathophysiology, CFTR function, and vector design and evaluation.

Read the original post:
Center for Gene Therapy - Research - University of Iowa

Concept

Genetic engineering is the alteration of genetic material by direct intervention in genetic processes with the purpose of producing new substances or improving functions of existing organisms. It is a very young, exciting, and controversial branch of the biological sciences. On the one hand, it offers the possibility of cures for diseases and countless material improvements to daily life. Hopes for the benefits of genetic engineering are symbolized by the Human Genome Project, a vast international effort to categorize all the genes in the human species. On the other hand, genetic engineering frightens many with its potential for misuse, either in Nazi-style schemes for population control or through simple bungling that might produce a biological holocaust caused by a man-made virus. Symbolic of the alarming possibilities is the furor inspired by a single concept on the cutting edge of genetic engineering: cloning.

How It Works

Dna

Any discussion of genetics makes reference to DNA (deoxyribonucleic acid), a molecule that contains genetic codes for inheritance. DNA resides in chromosomes, threadlike structures found in the nucleus, or control center, of every cell in every living thing. Chromosomes themselves are made up of genes, which carry codes for the production of proteins. The latter, of which there are many thousands of different varieties, make up the majority of the human body's dry weight.

Although it is central to the latest advances in modern genetic research, DNA was discovered more than 130 years ago. In 1869 the Swiss biochemist Johann Friedrich Miescher (1844-1895) isolated a substance, containing both nitrogen and phosphorus, that separated into a protein and an acid molecule. He called it nucleic acid, and in this material he discovered DNA. Some 74 years would pass, however, before scientists recognized the function of the nucleic acid Miescher had discovered. Then, in 1944, a research team led by the Canadian-born American bacteriologist Oswald Avery (1877-1955) found that by taking DNA from one type of bacterium and inserting it into another, the second bacterium took on certain traits of the first. This experiment, along with other experiments and research, proved that DNA serves as a blueprint for the characteristics and functions of organisms.

The Double Helix

Nine years later, in 1953, the American biochemist James D. Watson (1928-) and the English biochemist Francis Crick (1916-) solved the mystery of DNA's structure and explained the means by which it provides necessary instructions at critical moments in the course of cell division and growth. They proposed a double helix, or spiral staircase, model, which linked the chemical bases of DNA in definite pairs. Using this twisted-ladder model, they were able to explain how the DNA molecule could duplicate itself, since each side of the ladder is identical to the other; if separated, each would serve as the template for the formation of its mirror image.

The sides of the DNA ladder are composed of alternating sugar and phosphate molecules, like links in a chain, and consist of four different chemical bases: adenine, guanine, cytosine, and thymine. The four letters designating these basesA, G, C, and Tare the alphabet of the genetic code, and each rung of the DNA molecule is made up of a combination of two of these letters. Owing to specific chemical affinities, A always combines with T and C with G, to form what is called a base pair. Specific sequences of these base pairs, which are bonded to each other by atoms of hydrogen, constitute the genes.

Endless Combinations

See the original post here:
genetic engineering: Definition from Answers.com

Photo by: Gernot Krautberger

Genetic engineering is any process by which genetic material (the building blocks of heredity) is changed in such a way as to make possible the production of new substances or new functions. As an example, biologists have now learned how to transplant the gene that produces light in a firefly into tobacco plants. The function of that genethe production of lighthas been added to the normal list of functions of the tobacco plants.

Genetic engineering became possible only when scientists had discovered exactly what is a gene. Prior to the 1950s, the term gene was used to stand for a unit by which some genetic characteristic was transmitted from one generation to the next. Biologists talked about a "gene" for hair color, although they really had no idea as to what that gene was or what it looked like.

That situation changed dramatically in 1953. The English chemist Francis Crick (1916 ) and the American biologist James Watson (1928 ) determined a chemical explanation for a gene. Crick and Watson discovered the chemical structure for large, complex molecules that occur in the nuclei of all living cells, known as deoxyribonucleic acid (DNA).

DNA molecules, Crick and Watson announced, are very long chains or units made of a combination of a simple sugar and a phosphate group.

Amino acid: An organic compound from which proteins are made.

DNA (deoxyribonucleic acid): A large, complex chemical compound that makes up the core of a chromosome and whose segments consist of genes.

Gene: A segment of a DNA molecule that acts as a kind of code for the production of some specific protein. Genes carry instructions for the formation, functioning, and transmission of specific traits from one generation to another.

Gene splicing: The process by which genes are cut apart and put back together to provide them with some new function.

Genetic code: A set of nitrogen base combinations that act as a code for the production of certain amino acids.

See more here:
Genetic Engineering - humans, body, used, process, plants ...

Cogent Teaching Resources - Molecular Biology and Genetics

By: Cogent SSC

More here:
Cogent Teaching Resources - Molecular Biology and Genetics - Video

Early Flower Update/Ocean Grown Genetics
Bubble Krush, Sleeping Dog, Wizards Potion.

By: kbgrowkid420

Read the original:
Early Flower Update/Ocean Grown Genetics - Video

Best Response to Genetics Assignment
The assignment for Michelle Lawson #39;s science class was to creatively explain translation (the molecular process of translating mRNA into protein) on a single...

By: WillistonNorthampton

Read this article:
Best Response to Genetics Assignment - Video

keystone biology quick review genetic engineering

By: Jen DiPasquale

Read this article:
keystone biology quick review genetic engineering - Video

GROW YOUR OWN... at Science Gallery
Highlights of GROW YOUR OWN: LIFE AFTER NATURE at Science Gallery, Trinity College Dublin, October 2013. GROW YOUR OWN... is a new exhibition created by Scie...

By: Science Gallery

Read more here:
GROW YOUR OWN... at Science Gallery - Video

Genetics in Breast Cancer from BRCA to Tumor Profiling by Dr E Myburgh

By: Genetalk

Link:
Genetics in Breast Cancer from BRCA to Tumor Profiling by Dr E Myburgh - Video

Biology One Human Genetics
Patterns of Inheritance / mutation types.

By: MultiScienceclass

Continued here:
Biology One Human Genetics - Video

Report of the American Society of Human Genetics Ancestry Inference Roundtable - Charmaine Royal
September 12, 2013 - The African Diaspora: Integrating Culture, Genomics and History.

By: GenomeTV

Read more here:
Report of the American Society of Human Genetics Ancestry Inference Roundtable - Charmaine Royal - Video

Report of the American Society of Human Genetics Ancestry Inference Roundtable - Malia Fullerton
September 12, 2013 - The African Diaspora: Integrating Culture, Genomics and History.

By: GenomeTV

See the rest here:
Report of the American Society of Human Genetics Ancestry Inference Roundtable - Malia Fullerton - Video