Category Archives: Gene therapy

Gene therapy is the insertion of genes into an individual's cells and tissues to treat a disease, and hereditary diseases in which a defective mutant allele is replaced with a functional one.

Although the technology is still in its infancy, it has been used with some success.

Antisense therapy is not strictly a form of gene therapy, but is a genetically-mediated therapy and is often considered together with other methods.

In most gene therapy studies, a "normal" gene is inserted into the genome to replace an "abnormal," disease-causing gene.

A carrier called a vector must be used to deliver the therapeutic gene to the patient's target cells.

Currently, the most common type of vectors are viruses that have been genetically altered to carry normal human DNA.

Viruses have evolved a way of encapsulating and delivering their genes to human cells in a pathogenic manner.

Scientists have tried to harness this ability by manipulating the viral genome to remove disease-causing genes and insert therapeutic ones. Target cells such as the patient's liver or lung cells are infected with the vector.

The vector then unloads its genetic material containing the therapeutic human gene into the target cell.

The generation of a functional protein product from the therapeutic gene restores the target cell to a normal state. In theory it is possible to transform either somatic cells (most cells of the body) or cells of the germline (such as sperm cells, ova, and their stem cell precursors).

Excerpt from:
Gene therapy - Science Daily

Two adults with a rare disease that causes gradual loss of eyesight had their vision improved after being treated with a new gene therapy, according to preliminary results from a new study.

The study involved six patients ages 35 to 63 with choroideremia, an inherited condition with no cure that causes vision problems early in life, and eventually leads to blindness. Patients have a mutation in a gene called CHM, which causes light-sensitive cells in the eye to slowly stop working.

The goal behind the new gene therapy is to use a safe virus to deliver a working copy of the gene to the right part of the eye to prevent the cells from degenerating. [7 Diseases You Can Learn About From a Genetic Test]

The new study was an early test of the therapy in which the researchers aimed to carry out the treatment without causing damage to the eye. (Patients must have an eye surgery so that the virus can be injected under the retina with a fine needle).

The result showed that the treatment did not cause harm, and in fact, improved vision in a few of the patients.

Play Video

A UCLA eye surgeon has developed a possible stem cell treatment for Macular Degeneration, the leading cause of blindness in older Americans. Dr. ...

Six months after the treatment, four patients recovered the visual acuity (clearness or acuteness of vision) that they had before the surgery, and developed increased sensitivity to light. And two patients had improvements in vision: They were able to read two to four more lines on a sight chart.

"We did not expect to see such dramatic improvements in visual acuity," study researcher Robert MacLaren, of the Nuffield Laboratory of Ophthalmology at the University of Oxford in the U.K., said in a statement. It is still too early to know if the improvements will last, but they have so far been maintained for as long as two years, MacLaren said.

The study is the first to test gene therapy in patients before they'd experienced significant thinning of the retinal cells, MacLaren said.

More:
Gene therapy improves vision for some with rare disease

PUBLIC RELEASE DATE:

23-Dec-2013

Contact: Meng Zhao eic@nrren.org 86-138-049-98773 Neural Regeneration Research

In a recent study published in the Neural Regeneration Research (Vol. 8, No. 33, 2013), Prof. Feng Li and team from Zhongshan School of Medicine, Sun Yat-sen University in China, synthesized a 19-nt oligonucleotide targeting BACE1, the key enzyme in amyloid beta protein (A) production, and introduced it into the pSilenCircle vector to construct a short hairpin (shRNA) expression plasmid against the BACE1 gene. Researhcers transfected this vector into C17.2 neural stem cells and primary neural stem cells, resulting in downregulation of the BACE1 gene, which in turn induced a considerable reduction in reducing A protein production. This technique combining cell transplantation and gene therapy will open up novel therapeutic avenues for Alzheimer's disease, particularly because it can be used to simultaneously target several pathogenetic changes in the disease.

###

Article: " Targeting -secretase with RNAi in neural stem cells for Alzheimer's disease therapy " by Zhonghua Liu, Shengliang Li, Zibin Liang, Yan Zhao, Yulin Zhang, Yaqi Yang, Minjuan Wang, Feng Li (Department of Neurobiology and Anatomy, Zhongshan School of Medicine, Sun Yat-sen University, Guangzhou 510080, Guangdong Province, China)

Liu ZH, Li SL, Liang ZB, Zhao Y, Zhang YL, Yang YQ, Wang MJ, Li F. Targeting -secretase with RNAi in neural stem cells for Alzheimer's disease therapy. Neural Regen Res. 2013;8(33):3095-3106.

Contact: Meng Zhao eic@nrren.org 86-138-049-98773 Neural Regeneration Research http://www.nrronline.org/

Full text: http://www.sjzsyj.org/CN/article/downloadArticleFile.do?attachType=PDF&id=783

Excerpt from:
Combination of cell transplantation and gene therapy for Alzheimer's disease

-

The Center for Biologics Evaluation and Research (CBER) regulates cellular therapy products, human gene therapy products, and certain devices related to cell and gene therapy. CBER uses both the Public Health Service Act and the Federal Food Drug and Cosmetic Act as enabling statutes for oversight.

Cellular therapy products include cellular immunotherapies, and other types of both autologous and allogeneic cells for certain therapeutic indications, including adult and embryonic stem cells. Human gene therapy refers to products that introduce genetic material into a persons DNA to replace faulty or missing genetic material, thus treating a disease or abnormal medical condition.

Although some cellular therapy products have been approved, CBER has not yet approved any human gene therapy product for sale. However, the amount of cellular and gene therapy-related research and development occurring in the United States continues to grow at a fast rate. CBER has received many requests from medical researchers and manufacturers to study cellular and gene therapies and to develop cellular and gene therapy products. In addition to regulatory oversight of clinical studies, CBER provides proactive scientific and regulatory advice to medical researchers and manufacturers in the area of novel product development.

-

View original post here:
Cellular & Gene Therapy Products - Food and Drug Administration

Public release date: 9-Oct-2013 [ | E-mail | Share ]

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2100 x2156 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, October 9, 2013Cancer immunotherapy can successfully use the body's own immune system to kill tumor cells. But some current approaches to stimulate an antitumor immune response are short-lived, with limited clinical effectiveness. A new gene transfer strategy that introduces modified, immune-stimulating human stem cells is both feasible and effective for achieving persistent immunotherapy to treat leukemias and lymophomas, according to a study published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available on the Human Gene Therapy website.

Satiro Nakamura De Oliveira and coauthors from the David Geffen School of Medicine, University of California, Los Angeles and University of Texas MD Anderson Cancer Center, Houston, describe the gene transfer method they developed to deliver chimeric antigen receptors, or CARS, that direct the immune system to target tumor cells derived from B-lymphocytes.

In the article "Modification of Hematopoietic Stem/Progenitor Cells with CD19-specific Chimeric Antigen Receptors as a Novel Approach for Cancer Immunotherapy" the authors show that by packaging the CARS in human hematopoietic stem cells, the immunotherapeutic receptors will be produced in the bloodstream for a long period of time. This persistent expression should improve their effectiveness in the treatment of blood cancers such as leukemia and lymphoma.

"This study represents an interesting new direction for an approach that has generated substantial interest," says Dr. Wilson, Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia.

###

About the Journal

Human Gene Therapy, the official journal of the European Society of Gene and Cell Therapy, British Society for Gene and Cell Therapy, French Society of Cell and Gene Therapy, German Society of Gene Therapy, and five other gene therapy societies, is an authoritative peer-reviewed journal published monthly in print and online. Human Gene Therapy presents reports on the transfer and expression of genes in mammals, including humans. Related topics include improvements in vector development, delivery systems, and animal models, particularly in the areas of cancer, heart disease, viral disease, genetic disease, and neurological disease, as well as ethical, legal, and regulatory issues related to the gene transfer in humans. Its sister journal, Human Gene Therapy Methods, published bimonthly, focuses on the application of gene therapy to product testing and development, and Human Gene Therapy Clinical Development, new in 2013, publishes data relevant to the regulatory review and commercial development of cell and gene therapy products. Complete tables of content for all three publications and a free sample issue may be viewed on the Human Gene Therapy website.

About the Publisher

Original post:
Novel gene therapy enables persistent anti-tumor immune response

Shortly after a baby’s first wail at birth she or he receives a tiny prick on the heel. A few drops of blood are caught on special filter paper to screen for myriad diseases. [More]

-- Read more on ScientificAmerican.com

Source:
http://rss.sciam.com/~r/sciam/topic/gene-therapy/~3/qJICV6zfIAo/article.cfm

Four researchers from UCLA’s Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research have received Early Translational Research Awards totaling approximately $13 million from the California Institute for Regenerative Medicine, the state's stem cell agency. The UCLA researchers received four of the 12 total awards; no other institution received more than one.
 
The Independent Citizens Oversight Committee, CIRM’s governing body, announced at its Aug. 28 meeting in La Jolla, Calif., that grant recipients included Dr. Jerome Zack, professor of medicine and microbiology, immunology and molecular genetics; Dr. Robert Reiter, Bing Professor of Urologic Research; Dr. Donald Kohn, professor of pediatrics and microbiology, immunology and molecular genetics in th...

Source:
http://www.medworm.com/index.php?rid=7551755&cid=c_449_44_f&fid=38766&url=http%3A%2F%2Fnewsroom.ucla.edu%2Fportal%2Fucla%2Ffour-prominent-ucla-stem-cell-248077.aspx%3Flink_page_rss%3D248077

Four researchers from UCLA’s Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research have received Early Translational Research Awards totaling approximately $13 million from the California Institute for Regenerative Medicine, the state's stem cell agency. The UCLA researchers received four of the 12 total awards; no other institution received more than one.
 
The Independent Citizens Oversight Committee, CIRM’s governing body, announced at its Aug. 28 meeting in La Jolla, Calif., that grant recipients included Dr. Jerome Zack, professor of medicine and microbiology, immunology and molecular genetics; Dr. Robert Reiter, Bing Professor of Urologic Research; Dr. Donald Kohn, professor of pediatrics and microbiology, immunology and molecular genetics in th...

Source:
http://www.medworm.com/index.php?rid=7551755&cid=c_449_44_f&fid=38766&url=http%3A%2F%2Fnewsroom.ucla.edu%2Fportal%2Fucla%2Ffour-prominent-ucla-stem-cell-248077.aspx%3Flink_page_rss%3D248077

Scientists have come a step closer to being able to repair the damage done by heart attacks, using a cocktail of genes to transform scar tissue into working heart muscles.

Novel techniques to mend broken hearts using gene therapy and stem cells represent a major new frontier in the treatment of heart disease.

In the latest breakthrough, achieved by researchers at the Gladstone Institute of Cardiovascular Disease in California, researchers were able to re-programme scar-forming cells into heart muscle cells, some of which were capable of transmitting the kind of electrical signals that make the heart beat, according to the latest issue of the Stem Cell Reports journal.

The same team demonstrated their technique last year in live mice, transforming scar-forming cells, called fibroblasts, into beating heart muscle cells, but this is the first time that human fibroblasts have been re-programmed in this way.

So far, the work with human fibroblasts has only been done in the lab, but it paves the way for new treatments for heart attack victims. Researchers said that the cocktail of genes used to regenerate cells could one day be replaced with small drug-like molecules that would offer safer and easier delivery.

We've now laid a solid foundation for developing a way to reverse the damage [done by a heart attack] something previously thought impossible and changing the way that doctors may treat heart attacks in the future, said Dr Deepak Srivastava, director of cardiovascular disease at the Gladstone Institutes. Our findings here serve as a proof of concept that human fibroblasts can be re-programmed successfully into beating heart cells.

In 2012, Dr Srivastava and his team reported in the journal Nature that, by injecting three genes into the hearts of live mice that had been damaged by heart attack, fibroblasts could be turned into working heart cells.

The scientists attempted the same technique using human fibroblasts from foetal heart cells, embryonic stem cells and neonatal skin cells, injected with genes in petri dishes in the lab. An increased number of genes was required to transform the human cells, and the efficiency of the transformed cells was low, but the team were encouraged by the results.

While almost all the cells in our study exhibited at least a partial transformation, about 20 per cent of them were capable of transmitting electrical signals a key feature of beating hearts, said Gladstone staff scientist Ji-dong Fu, the studys lead author.

The number of people who survive heart attacks has increased considerably in recent decades. The British Heart Foundation (BHF) said earlier this year that 70 per cent of women and 68 per cent of men were now surviving. However, success in keeping people alive after a heart attack has led to a rise in the number of people suffering from the long-term after-effects, which include debilitating heart failure.

See original here:
Scientists use gene therapy to repair muscles damaged in heart attacks

Scientists have come a step closer to being able to repair the damage done by heart attacks, using a cocktail of genes to transform scar tissue into working heart muscles.

Novel techniques to mend broken hearts using gene therapy and stem cells represent a major new frontier in the treatment of heart disease.

In the latest breakthrough, achieved by researchers at the Gladstone Institute of Cardiovascular Disease in California, researchers were able to re-programme scar-forming cells into heart muscle cells, some of which were capable of transmitting the kind of electrical signals that make the heart beat, according to the latest issue of the Stem Cell Reports journal.

The same team demonstrated their technique last year in live mice, transforming scar-forming cells, called fibroblasts, into beating heart muscle cells, but this is the first time that human fibroblasts have been re-programmed in this way.

So far, the work with human fibroblasts has only been done in the lab, but it paves the way for new treatments for heart attack victims. Researchers said that the cocktail of genes used to regenerate cells could one day be replaced with small drug-like molecules that would offer safer and easier delivery.

We've now laid a solid foundation for developing a way to reverse the damage [done by a heart attack] something previously thought impossible and changing the way that doctors may treat heart attacks in the future, said Dr Deepak Srivastava, director of cardiovascular disease at the Gladstone Institutes. Our findings here serve as a proof of concept that human fibroblasts can be re-programmed successfully into beating heart cells.

In 2012, Dr Srivastava and his team reported in the journal Nature that, by injecting three genes into the hearts of live mice that had been damaged by heart attack, fibroblasts could be turned into working heart cells.

The scientists attempted the same technique using human fibroblasts from foetal heart cells, embryonic stem cells and neonatal skin cells, injected with genes in petri dishes in the lab. An increased number of genes was required to transform the human cells, and the efficiency of the transformed cells was low, but the team were encouraged by the results.

While almost all the cells in our study exhibited at least a partial transformation, about 20 per cent of them were capable of transmitting electrical signals a key feature of beating hearts, said Gladstone staff scientist Ji-dong Fu, the studys lead author.

The number of people who survive heart attacks has increased considerably in recent decades. The British Heart Foundation (BHF) said earlier this year that 70 per cent of women and 68 per cent of men were now surviving. However, success in keeping people alive after a heart attack has led to a rise in the number of people suffering from the long-term after-effects, which include debilitating heart failure.

More:
Scientists come closer to 'mending broken hearts' by using gene therapy to repair muscles damaged in heart attacks