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Category Archives: Gene Medicine

San Francisco: 41-year-old black rhino 'Gene' dies zoo

SAN FRANCISCO --San Francisco zoo officials are mourning the death of "Gene," a 41-year-old black rhinoceros who's been a popular figure at the zoo since 1978.

Zoo officials, who described Gene as gentle and friendly, said he died of kidney failure on Monday.

The zoo's animal care and veterinary staff had been keeping an eye on Gene because his appetite had dropped and he was lethargic, according to zoo officials.

After a recent blood sample indicated that Gene had kidney failure, zoo staff focused on keeping him comfortable for the remainder of his life.

Gene was named after the late Eugene Friend, who served on the Recreation and Park Commission for 24 years, zoo officials said.

Gene was born in Kenya and came to the San Francisco Zoo in 1978 at the age of seven.

During his time at the zoo, Gene fathered five offspring, three of which are now living at other accredited zoos, zoo officials said.

Gene's animal keeper, Julie McGilvray, said he had a good disposition and was very friendly.

"We nicknamed him Big Dog because he loved to be rubbed, either by hand or with a scrub brush, and oftentimes he would lie down and absorb the soothing experience," McGilvray said in a statement.

Zoo officials said black rhinos are a critically endangered species because they are targeted by poachers who covet their valuable horns.

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41-year-old black rhino 'Gene' dies of kidney failure

SAN FRANCISCO -- San Francisco Zoo officials are mourning the death of "Gene," a 41-year-old black rhinoceros who's been a popular figure at the zoo since 1978.

Zoo officials, who described Gene as gentle and friendly, said he died of kidney failure on Monday.

The zoo's animal care and veterinary staff had been keeping an eye on Gene because his appetite had dropped and he was lethargic, according to zoo officials.

After a recent blood sample indicated that Gene had kidney failure, zoo staff focused on keeping him comfortable for the remainder of his life.

Gene was named after the late Eugene Friend, who served on the Recreation and Park Commission for 24 years, zoo officials said.

Gene was born in Kenya and came to the San Francisco Zoo in 1978 at the age of seven.

During his time at the zoo, Gene fathered five offspring, three of which are now living at other accredited zoos, zoo officials said.

Gene's animal keeper, Julie McGilvray, said he had a good disposition and was very friendly.

"We nicknamed him Big Dog because he loved to be rubbed, either by hand or with a scrub brush, and oftentimes he would lie down and absorb the soothing experience," McGilvray said in a statement.

Zoo officials said black rhinos are a critically endangered species because they are targeted by poachers who covet their valuable horns.

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Workshop 5: Genetics of Complex Disease – Joan Esnayra – Video

08-02-2012 12:15 Joan shares her family story of bipolar disorder and her journey as a mental health advocate. This workshop is part of a genomics curriculum for practicing healthcare providers developed by the Genomic Medicine Institute at El Camino Hospital, Genetic Alliance, and the National Coalition for Health Professional Education in Genetics. This workshop, the fifth in a 10-part series, covered understanding genetic contribution to schizophrenia, major depression, hypertension, and diabetes; risk assessment for complex conditions; establishing diagnosis; pharmacogenomics; gene-environment interaction; and management.

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Bladder Pain Syndrome – Gene Expression Analysis Shows Promise

Editor's Choice Academic Journal Main Category: Urology / Nephrology Article Date: 28 Feb 2012 - 9:00 PST

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Interstitial cystitis or bladder pain syndrome is a debilitating urinary bladder disease that can occur with or without bladder ulcers - called Hunner lesions. Researching the disease is difficult, due to limited animal models, because human patients are not ethically permitted to undergo invasive research procedures.

The researchers set out to develop a noninvasive method to evaluate the bladder epithelium as objectively and directly as possible by using microarray technology to examine cells shed into the urine. The alternative would be conducting a bladder biopsy, which requires anesthesia and has a small risk of injuring the bladder.

The findings demonstrated that urine cells, from patients with Hunner lesions had a distinct gene signature for inflammation. The findings were comparable to those from an earlier microarray study of bladder biopsies, which was the first to objectively demonstrate this inflammation without biopsy in Hunner lesion patients.

Eric Blalock, associate professor in the Department of Molecular and Biomedical Pharmacology at the UK College of Medicine, says that providing these preliminary results validate future research, they could potentially lead to a noninvasive biomarker for Hunner lesion-interstitial cystitis/bladder pain syndrome.

He states:

Gene signatures were similar to healthy controls in interstitial cystitis patients without Hunner lesions.

Urologist Deborah Erickson declared:

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Bladder Pain Syndrome - Gene Expression Analysis Shows Promise

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Global Gene Therapy Market to Reach US$794 Million by 2017, According to New Report by Global Industry Analysts, Inc.

GIA announces the release of a comprehensive global report on Gene Therapy markets. The global market for Gene Therapy is forecast to reach US$794 million by 2017. Key factors driving growth in the market include rising demand for new and effective therapies for cancer treatment, in addition to completion of human genome project, rising incidence and prevalence of cancers and other critical diseases, prospective launch of gene therapies in major global markets.

San Jose, California (PRWEB) February 28, 2012

Follow us on LinkedIn – Gene therapy embarks on rapid advancements in the area of novel drug research. In less than a couple of decades, gene therapy has witnessed significant advances. From conceptual stage, gene therapy progressed to the current clinical trials stage in various disease conditions. Gene Therapy holds a distinctive edge when compared to conventional methods such as radiotherapy and chemotherapy, owing to the fact that they do not repress the immune system. Analysis reveals that gene therapy holds a great potential for treating several diseases, especially cancer. However, with failures of gene therapies continuing worldwide and regulatory authorities being stringent and watchful, and no drug approved until now in major markets like US and Europe, the global gene therapy market has significant ground to cover. Scientists, besides being actively involved in expanding the horizons in gene therapy by research and development, are simultaneously involved in patenting new technologies.

The most important advantage of gene therapy is elimination of chemotherapy, which is a dreaded procedure with several side effects. Another advantage of gene therapy is evasion of several side effects including hearing impairment and kidney damage resulting from platinum-induced chemotherapy. Gendicine is the first gene therapy medicine to be approved for treatment of Head and Neck Squamous Cell Carcinoma (HNSCC), and is currently marketed in China. The drug developed by Shenzhen SiBiono Gene Technologies Co (SiBiono) Ltd., is also called as Recombinant Ad-p53 Anti-cancer Injection. The medicine is formulated with an adenoviral vector and p53 tumor suppressor gene and has emerged as a new treatment for HNSCC. Worldwide, there are about five drugs that have completed clinical trials and are awaiting clearance from the concerned regulatory authorities. These drugs include HGF DNA Plasmid from Sosei and Daiichi Sankyo. The drug investigated for treating peripheral vascular disease was filed for approval in Japan way back in 2008, and is yet to receive approval from the Japanese authorities.

Regulation of gene therapy needs to be harmonized on a global scale for promoting international exchange. This could be achieved by extensive circulation of information. With rapid advancements made in the field of gene therapy, the regulation norms tend to change as storehouse of knowledge increases. Somatic gene therapy encompasses a broad spectrum of therapeutic strategies and procedures; hence, the review process is being conducted in a phased manner. Currently under clinical trials, gene therapy is far from being stabilized. Steps need to be taken for regulation of gene therapy with respect to safety features. Development of gene therapy is hampered due to lack of efficiency and specificity of Gene Transfer Systems. High levels of uncertainty envelop technical, clinical, and commercial development of gene therapy.

Major players profiled in the report include AnGes MG Inc., BioSante Pharmaceuticals, GenVec, Oxford BioMedica, Shenzhen SiBiono GeneTech Co., Ltd, Transgene, Urigen Pharmaceuticals Inc., and Vical, Inc.

The research report titled “Gene Therapy: A Global Strategic Business Report” announced by Global Industry Analysts Inc., provides a comprehensive review of the gene therapy market, current market trends, key growth drivers, recent industry activity, and profiles of major/niche global market participants. The report provides annual sales estimates and projections for global gene therapy market for the years 2008 through 2017.

For more details about this comprehensive market research report, please visit –

http://www.strategyr.com/Gene_Therapy_Market_Report.asp

About Global Industry Analysts, Inc.

Global Industry Analysts, Inc., (GIA) is a leading publisher of off-the-shelf market research. Founded in 1987, the company currently employs over 800 people worldwide. Annually, GIA publishes more than 1300 full-scale research reports and analyzes 40,000+ market and technology trends while monitoring more than 126,000 Companies worldwide. Serving over 9500 clients in 27 countries, GIA is recognized today, as one of the world's largest and reputed market research firms.

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Telephone: 408-528-9966

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Antisense oligonucleotides make sense in myotonic dystrophy

Public release date: 27-Feb-2012
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Contact: Graciela Gutierrez
ggutierr@bcm.edu
713-798-4710
Baylor College of Medicine

HOUSTON (Feb. 27, 2012) ? Antisense oligonucleotides ? short segments of genetic material designed to target specific areas of a gene or chromosome ? that activated an enzyme to "chew up" toxic RNA (ribonucleic acid) could point the way to a treatment for a degenerative muscle disease called myotonic dystrophy, said researchers from Baylor College of Medicine and Isis Pharmaceuticals, Inc., in a report in the journal Proceedings of the National Academy of Sciences.

"This is a proof-of-principle therapy that is very effective in cell culture and mice," said Dr. Thomas A. Cooper, professor of pathology and immunology and molecular and cellular biology at BCM and the report's corresponding author. "The treatment will have to be refined to deliver systemically in people with myotonic dystrophy."

Myotonic dystrophy is the most common muscular disease in adults, affecting mainly the skeletal muscles, heart and central nervous system. It occurs because of a mutation that causes numerous repeats of three letters of the genetic code (CTG) in a gene called DMPK. RNA is made as a step in the cell's production of the protein associated with the gene. The messenger RNA (the chemical blueprint for making a protein) that is produced from the mutated gene also contains the abnormal long repeats that cause the RNA to accumulate in the cell's nucleus. There it sequesters and blocks the function of a protein called Muscleblind-like 1 and activates another protein called CELF1. These proteins antagonize one another and the result is abnormal expression of proteins from many other genes in adult tissues, resulting in disease.

To counteract this, Cooper and his colleagues created antisense oligonucleotides called gapmers, which are simply strands of genetic material that seek out portions of the abnormal RNA repeats and target an enzyme called RNase H to the toxic RNA causing its degradation. They also showed that combining the gapmers with other antisense oligonucleotides that help released the sequestered Muscleblind-like1 can enhance the effect.

"It worked in cultures of cells with the expanded repeats and in mice that model myotonic dystrophy," said Cooper. "We did it in skeletal muscle first because we can inject the material directly into the muscle."

Later, he plans to determine if the material also works in the animals' hearts.

Using the treatment in people will require more fine-tuning, said Cooper. He would like to be able to give the therapy systemically rather than directly into the muscle. They saw some muscle damage and inflammation in the animals they treated.

Antisense oligonucleotide treatments are being tested in Duchenne muscular dystrophy and another disease called spinal muscular atrophy, said Cooper.

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Others who took part in this research include Johanna E. Lee of BCM and C. Frank Bennett of Isis.

Funding for this work came from the National Institutes of Health, the Muscular Dystrophy Association and the Shanna and Andrew Linbeck Family Charitable Fund.

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Antisense oligonucleotides make sense in myotonic dystrophy

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