Category Archives: Gene therapy

Earlier this year Yaniv Erlich of the Whitehead Institute for Biomedical Research at M.I.T. sent bioethicists into a frenzy when he and his team uncovered the names of people whose anonymous genome...

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Macular Degeneration And Acupuncture: Gene Therapy and Stem Cell for AMD Safe?
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Macular Degeneration And Acupuncture: Gene Therapy and Stem Cell for AMD Safe? - Video

July 24, 2013 Scientists from The University of Manchester have used stem cell gene therapy to treat a fatal genetic brain disease in mice for the first time.

The method was used to treat Sanfilippo -- a fatal inherited condition which causes progressive dementia in children -- but could also benefit several neurological, genetic diseases.

Researchers behind the study, published in the journal Molecular Therapy this month, are now hoping to bring a treatment to trial in patients within two years.

Sanfilippo, a currently untreatable mucopolysaccharide (MPS) disease, affects one in 89,000 children in the United Kingdom, with sufferers usually dying in their mid-twenties. It is caused by the lack of SGSH enzyme in the body which helps to breakdown and recycle long chain sugars, such as heparan sulphate (HS). Children with the condition build up and store excess HS throughout their body from birth which affects their brain and results in progressive dementia and hyperactivity, followed by losing the ability to walk and swallow.

Dr Brian Bigger, from the University of Manchester's Institute of Human Development who led the research, said bone marrow transplants had been used to correct similar HS storage diseases, such as Hurler syndrome, by transplanting normal cells with the missing enzyme but the technique did not work with Sanfilippo disease. This is because monocytes, a type of white blood cell, from the bone marrow, did not produce enough enzyme to correct the levels in the brain.

Dr Bigger said: "To increase SGSH enzyme from bone marrow transplants, and to target it to the cells that traffic into the brain, we have developed a stem cell gene therapy which overproduces the SGSH enzyme specifically in bone marrow white blood cells. "We have shown that mice treated by this method produce five times the normal SGSH enzyme levels in the bone marrow and 11 per cent of normal levels in the brain.

"The enzyme is taken up by affected brain cells and is enough to correct brain HS storage and neuro inflammation to near normal levels and completely corrects the hyperactive behaviour in mice with Sanfilippo.

"This is extremely exciting and could have huge implications for treatments. We now hope to work to a clinical trial in Manchester in 2015."

The University of Manchester team is now manufacturing a vector -- a tool commonly used by molecular biologists to deliver genetic material into cells -- for use in humans and hope to use this in a clinical trial with patients at Central Manchester University Hospital NHS Foundation Trust by 2015.

The stem cell gene therapy approach was recently shown by Italian scientists to improve conditions in patients with a similar genetic disease affecting the brain called metachromatic leukodystrophy, with results published in the journal Science earlier this month. Manchester scientists refined the vector used by the Italian scientists. "This approach has the potential to treat several neurological genetic diseases," Dr Bigger added.

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New stem cell gene therapy gives hope to prevent inherited neurological disease

NEW YORK, July 22, 2013 /PRNewswire/ -- Reportlinker.com announces that a new market research report is available in its catalogue:

Global Gene Therapy Market Analysis

http://www.reportlinker.com/p0324817/Global-Gene-Therapy-Market-Analysis.html#utm_source=prnewswire&utm_medium=pr&utm_campaign=Biological_Therapy

Gene therapy is the treatment of a disease by replacing, altering, or supplementing a gene that is absent or abnormal and whose absence or abnormality is causing the disease. It has evolved as one of the most sought after research objectives for 'difficult to cure' diseases. The gene therapy market in spite of presenting few marketable products and being nascent in terms of revenue generation holds tremendous growth potential. As per a new estimation carried out in our latest study, the global gene therapy industry has the potential to become a multi-million dollar industry by the end of 2017 as new products, especially those in the advanced stage of clinical studies or with pending approvals, may enter the market to boost the growth.

According to RNCOS' new research report, "Global Gene Therapy Market Analysis", major focus has been made on the ongoing clinical trials for the development of innovative products. Majority trials are focused on oncology and they prove promising on global scale for US. In this context, the study provides a comprehensive overview of the various aspects of clinical trials in the gene therapy market such as phases, geographies, vector types etc.

The report provides an in-depth and prudent analysis of the industry and research developments taking place at the global level. Primarily the market is presently dominated by oncology applications with several companies and academic institutions focusing on novel and 'difficult to treat' cancers. Other therapeutic areas seeing developments on gene therapy include cardiac conditions, genetic disorders and neurological diseases. An effective analysis of the key therapeutic areas has been included in the report.

Brief overview of the key geographies in the gene therapy market and the regulatory scenario governing the industry has also been included in the study to present a balanced outlook. The report covers how major trends and drivers, including gene silencing, advanced therapies etc will propel the industry's growth. With a view to understanding the competitive landscape, the profiles of key market players are included in the report to present a complete picture of the global gene therapy market.1. Analyst View2. Research Methodology3. Gene Therapy - An Introduction3.1 Classification of Gene Therapy Techniques3.2 Physical Methods of Gene Transfer3.2.1 Electroporation3.2.2 Hydrodynamic3.2.3 Microinjection3.2.4 Particle Bombardment3.2.5 Ultrasound-mediated Transfection3.3 Vectors for Gene Therapy3.3.1 Adenoviral Vectors3.3.2 Adeno-associated Virus Vectors3.3.3 Retroviral Vectors3.3.4 Lentiviral Vectors3.3.5 Plasmid DNA4. Industry Trends and Drivers4.1 Gene Silencing: Gaining Momentum4.2 Advanced Therapies: Combining Gene Therapy and Stem Cell Technology4.3 Immunodeficiency Syndromes: The Focus of Gene Therapy4.4 Nanotechnology: Empowering Gene Therapy4.5 Gene Therapy: A New Hope to Treat Blindness5. Gene Therapy Clinical Trial Assessment5.1 By Geography5.2 By Indication5.3 By Gene Type5.4 By Vector5.5 By Clinical Trial Phase6. Industry Overview6.1 Market Size and Growth6.2 By Application6.2.1 Oncology6.2.1.1 Key Approaches6.2.1.2 Recent Advancements6.2.2 Infectious Diseases6.2.2.1 Key Approaches6.2.2.2 Recent Advancements6.2.3 Genetic Disorders6.2.3.1 Key Approaches6.2.3.2 Recent Advancements6.2.4 Cardiovascular Disorders6.2.4.1 Key Approaches6.2.4.2 Recent Advancements6.2.5 Diabetes Mellitus6.2.5.1 Key Approaches6.2.5.2 Recent Advancements6.2.6 Neurological Disorders6.2.6.1 Key Approaches6.2.6.2 Recent Advancements6.3 Key Geographical Markets6.3.1 US6.3.1.1 Regulatory Landscape6.3.1.2 Recent Advancements6.3.2 Europe6.3.2.1 Regulatory Landscape6.3.2.2 Recent Advancements6.3.3 Australia6.3.3.1 Regulatory Landscape6.3.3.2 Recent Advancements6.3.4 Canada6.3.4.1 Regulatory Landscape6.3.4.2 Recent Advancements6.3.5 Japan6.3.5.1 Regulatory Landscape6.3.5.2 Recent Advancements6.3.6 China6.3.6.1 Regulatory Landscape6.3.7 India6.3.7.1 Regulatory Landscape6.3.7.2 Recent Advancements6.4 Advanced Gene Therapy Drugs6.4.1 Collategene6.4.2 Allovectin-76.4.3 Generx7. Key Players Analysis7.1 AnGes MG, Inc7.2 Ark Therapeutics7.3 Ceregene, Inc7.4 Cobra Biologics7.5 GenVec, Inc7.6 Genzyme Corporation7.7 Vical, Inc7.8 Oxford BioMedica

List of Figures:

Figure 5-2: Percentage of Gene Types Transferred in Gene Therapy Trials till 2012

Figure 5-3: Percentage of Vectors Used in Gene Therapy Trials till 2012

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Global Gene Therapy Market Analysis

Abstract
Few options are available for treating patients with advanced prostate cancer (PC). As PC is a slow growing disease and accessible by ultrasound, gene therapy could provide a viable option for this neoplasm. Conditionally replication?competent adenoviruses (CRCAs) represent potentially useful reagents for treating prostate cancer (PC). We previously constructed a CRCA, Cancer Terminator Virus (CTV), which showed efficacy both in vitro and in vivo for PC. The CTV was generated on a serotype 5?background (Ad.5?CTV) with infectivity depending on Coxsackie?Adenovirus Receptors (CARs). CARs are frequently reduced in many tumor types, including PCs thereby limiting effective Ad?mediated therapy. Using serotype chimerism, a novel CTV (Ad.5/3?CTV) was created by replacing the ...

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Two rare hereditary disorders, one of which kills children within the first few years of life, can be treated with gene therapy, new research from Italy suggests.

In children with the disorders, those who received gene therapy in which a "faulty" gene is replaced with a healthy one showed either improvement in their symptoms or a halt in the disease's progression. The children did not appear to experience serious side effects resulting from the gene therapy.

One disorder, called metachromatic leukodystrophy, causes a buildup of fatty acids in the brain, which leads to cognitive and movement problems and, ultimately, death at an early age.

The researchers treated three children with genetic mutations for metachromatic leukodystrophy, all of whom had older siblings with the condition. Because the patients were very young, ages 7 to 15 months at the study's start, they did not show full symptoms of the condition.

By age 3, one of the children treated with the gene therapy had a normal IQ score and language skills for his or her age, and was able to stand up voluntarily and walk holding someone's hand. In contrast, siblings of this patient who did not receive the therapy were incapable of speech and wheelchair bound by age 3.

The two other patients with the condition, who were also treated with gene therapy, did not show symptoms by age 2, an age at which researchers would have expected symptoms to appear.

Gene therapy was also used to treat three children with Wiskott-Aldrich syndrome, an immune system disorder caused by mutations in a gene called WAS. People with this condition are at increased risk for developing infections, as well as eczema. The children treated with the gene therapy saw their symptoms decrease or disappear within 20 to 30 months of undergoing treatment, the researchers said.

Though the results are promising, the study period was relatively short, and researchers said they need to continue to monitor all six children for changes in their conditions. [9 Most Bizarre Medical Conditions ]

Both groups of children (those with metachromatic leukodystrophy, and those with Wiskott-Aldrich syndrome) received very similar gene-therapy treatments. The researchers removed blood stem cells, called hematopoietic stem cells, from the patients, and used a virus to introduce a corrected form of each patient's faulty gene. These cells were then infused back into the patients.

In patients with Wiskott-Aldrich syndrome, blood stem cells are directly affected by the disease, so the newly infused stem cells replace the diseased cells, the researchers said. For patients with metachromatic leukodystrophy, the newly infused stem cells find their way to the brain, where they release the corrected form of the gene product (a protein), which, in turn, is taken in by the brian cells.

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6 children with rare disorders helped by gene therapy

Gene therapy: not just for mice.

Genetic traits, like a bulbous nose or balding, give some people reasons to moan about what they inherited from their parents. But more serious genetic flaws can cause debilitating disease. Now, Italian researchers have come up with a way of treating one such inherited disease and reversing another using a promising new method of gene therapy.

The idea behind gene therapy is to replace a faulty gene with a shiny new version that works properly. Modified versions of viruses, which have been sculpted by millions of years of evolution, perfectly penetrate human cells. They act as courriers delivering DNA payloads to defective cells and ensure they are stably inherited.

This deceptively simple idea, though, has been challenging to achieve in practice. The first commercial gene therapy product, Glybera, only received regulatory approval in 2012.

Part of the reason for this is the difficulty of successfully clearing three hurdles at once: delivering replacement genetic information to the exact cells that need help, getting this information safely translated in high enough volumes to overcome the defects, and stopping the immune system from reacting to normal genes when it has grown used to only seeing mangled ones.

Now, a team led by Alessandra Biffi at the San Raffaele Scientific Institute in Milan, Italy, reports inScience that they have developed a new approach that navigates each of these hurdles to treat three children with metachromatic leukodystrophy (MLD), a devastating inherited disease that affects around 1 in 40,000 people.

MLD usually manifests in early childhood and kills patients just a few years after the first symptoms appear. It is caused by a defect in a single gene, ARSA. This gene encodes information used by the lysosome, a piece of recycling machinery used by human cells to break down unwanted material. When this recycling process does not work properly in nerve cells, as is the case with MLD patients, they become filled with rubbish and begin to slowly decline, leading to brain and spinal cord degeneration, as well as sensory deprivation.

Supplying a replacement ARSA gene to affected cells in the nervous system is a tricky task, because these areas are heavily protected. To overcome these defenses, the team employed haematopoietic stem cells (HSCs), which can usually be found nestling quietly in the bone marrow, as stealthy genetic courriers. A tiny number of these cells were harvested from each patient, loaded with benign viruses carrying a working copy of ARSA, and put back into the bloodstream.

These engineered cells either lodged in the bone marrow or continued to travel around the body in the blood, where they corrected defective cells in the nervous system by supplying the normal version of ARSA. Because these were stem cells, they also reproduced to form new blood cells that themselves took on the same supportive roles.

Most MLD patients produce a garbled version of ARSA that has a very low level of activity, nowhere near enough to let the lysosome carry out its normal job. Restoring partial activity is not enough to make a clinical impactlevels must be hiked to make an obvious difference.

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Two successful gene therapy trials block inherited diseases in humans

A new type of gene therapy has shown promise in wiping out two rare childhood diseases, apparently without the risks of causing cancer, international researchers said Thursday.

The method used an HIV virus vector and the patients' own blood stem cells to deliver a corrected version of a faulty gene, said the report in the US journal Science.

As a result, six children are doing well, 18 to 32 months after their operations, said lead scientist Luigi Naldini of the San Raffaele Telethon Institute for Gene Therapy in Milan.

"Three years after the start of the clinical trial, the results obtained from the first six patients are very encouraging. The therapy is not only safe, but also effective and able to change the clinical history of these severe diseases."

Three of the children suffer from metachromatic leukodystrophy, a disease of the nervous system which is caused by mutations in the ARSA gene.

Babies with the disease appear healthy when they are born, but as they grow, they start to lose cognitive and motor skills. There is no cure.

The new gene therapy approach has halted progression of the disease in these three children, researchers said.

The other three children in the study have Wiskott-Aldrich syndrome, which is caused by mutations in the WAS gene and results in recurring infections, autoimmune diseases, frequent bleeding, and a high risk of cancer.

The treatment has caused the children's symptoms to lessen or vanish altogether, the researchers said.

Previous attempts at gene therapy for a range of diseases, including Wiskott-Aldrich Syndrome, have shown some success, but over the long term it was discovered that immune-compromised patients sometimes developed blood cancer.

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New gene therapy hope for rare childhood disease

July 11, 2013 Two Houston researchers from Baylor College of Medicine and Texas Children's Hospital were part of an international team that developed a new gene therapy approach to treatment of Wiskott-Aldrich Syndrome, a fatal inherited form of immunodeficiency.

The new research, led by researchers at the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy was published in Science Express.

The disorder that weakens the body's immune system is caused by a mutation in a gene that encodes the protein WASP. The most often used therapy is a bone marrow or stem cell transplant from a matching donor -- often a sibling or relative. It can be curative for some patients -- mostly those who have a strongly matching donor.

An alternative is to obtain blood stem cells from patient, and, in the laboratory, use gene therapy involving a form of retrovirus to take the normal gene into the cells to correct the defect. The patients are then given back the genetically changed blood cells back.

This approach has been successful in a number of diseases, including who had Wiskott-Aldrich Syndrome. However, over the long term, some patients with immune deficiencies, including those with Wiskott-Aldrich Syndrome, developed blood cancers.

Researchers believe the viral vector used to take the gene into the cell inserted itself next to a oncogene in the DNA, turning it on and causing the cancers.

In this new research, the team used a partially inactivated lentivirus to take the normal gene into the cell, while reducing the risk of the gene inserting next to a cancer-promoting gene.

In these cases for whom there was no matching donor, the researchers led by Alessandro Aiuti of San Raffaele Telethon Institute for Gene Therapy in Milan, Italy; and including Dr. Jordan Orange, professor of pediatrics--rheumatology and Pinaki Banerjee, assistant professor of pediatrics -- human immunology at BCM and Texas Children's, took the patients' own blood stem cells and, in the laboratory, used the lentiviral vector combined with the normal WASP gene to correct the genetic defect in the blood. After a special treatment to eliminate their defective immune system, they received their own blood cells that had been altered to contain the normal WASP gene.

After 20 to 30 months, the three children showed significant improvement. New blood cells had the corrected WASP gene.

Orange referred one of the patients for treatment and saw one of them at a recent meeting. He and Banerjee contributed to the analysis of the gene corrected patient cells at the laboratory level using highly quantitative high-resolution imaging Orange maintains a high-and super resolution imaging facility in the Center for Human Immunobiology at Texas Children's Hospital.

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Gene therapy using lentivirus to treat Wiskott-Aldrich Syndrome promising

WASHINGTON: A new type of gene therapy has shown promise in wiping out two rare childhood diseases, apparently without the risks of causing cancer, international researchers said Thursday.

The method used an HIV virus vector and the patients' own blood stem cells to deliver a corrected version of a faulty gene, said the report in the US journal Science.

As a result, six children are doing well, 18 to 32 months after their operations, said lead scientist Luigi Naldini of the San Raffaele Telethon Institute for Gene Therapy in Milan.

"Three years after the start of the clinical trial, the results obtained from the first six patients are very encouraging. The therapy is not only safe, but also effective and able to change the clinical history of these severe diseases."

Three of the children suffer from metachromatic leukodystrophy, a disease of the nervous system which is caused by mutations in the ARSA gene.

Babies with the disease appear healthy when they are born, but as they grow, they start to lose cognitive and motor skills. There is no cure.

The new gene therapy approach has halted progression of the disease in these three children, researchers said.

The other three children in the study have Wiskott-Aldrich syndrome, which is caused by mutations in the WAS gene and results in recurring infections, autoimmune diseases, frequent bleeding, and a high risk of cancer.

The treatment has caused the children's symptoms to lessen or vanish altogether, the researchers said.

Previous attempts at gene therapy for a range of diseases, including Wiskott-Aldrich Syndrome, have shown some success, but over the long term it was discovered that immune-compromised patients sometimes developed blood cancer.

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New gene therapy hope for rare childhood diseases