Gene Therapy

New Line Genetics and Offer $5,000 for Your DNA

by Dr. Hsien-Hsien Lei
Posted October 29, 2007 in DNA Testing, DNA in General, Gene Therapy, Polls About DNA

cold spring harbor dnaOver the past few weeks, I’ve been communicating with Anthony Martin, the founder and owner of New Line Genetics, a Silicon Valley company positioned at the forefront of the genomics revolution. He’s got some interesting and innovative ideas that will definitely get people talking. I’ve invited him to introduce his company today. (Disclaimer: I have no financial interest in New Line Genetics.)

Hsien has graciously offered me the space for a guest post today, and I am honored to accept. I would like to use this platform to announce the official launch of! This site will allow any individual to sell his or her DNA to New Line Genetics for research and development purposes, and each DNA donor will be paid at least $5,000 USD for their sample.

Since I launched the company back in 2002, we’ve invested hundreds of millions of dollars into genetics research, and we’ve grown at a 50% rate in both profits and staff size every year. With the enormous revenue growth we expect from our proprietary organ-farming system, we are poised to become the largest and most influential genetics company in the world within the next five years. During this time we plan to vastly improve the current state of medical genetics, and use our developing cybernetics technology to lay the groundwork for the next stage of human evolution.

In order to continue at our current pace of research and development, we need your help. Our existing cell lines are no longer sufficient for the volume of work we’re doing, so we’ve been looking into ways to obtain viable samples in a cost-effective and legal way. To this end, New Line Genetics is leading a pilot program with the patent office that allows us to patent individual genomes. This new process circumvents all current restrictions on sequence patents, since we are patenting a totally unique sequence, not a generic one. As long as the individual donor is aware that his genomic sequence will no longer belong to him, and said donor receives appropriate payment for goods sold, we are free to use the DNA sample in any of our existing programs.

This is good news for both us and you. We will be able to continue our groundbreaking research, and you’ll be able to earn at least $5,000 USD by selling something you don’t even use! As an added bonus to you, if your specific DNA sample is used to generate a replacement organ that we grow in our lab and sell to a hospital, you will be eligible to receive a percentage of the profit. So please, tell your friends and family about, and keep those samples coming in!

Would you sell your DNA? Take the poll below the fold!

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Exon Skipping Gene Therapy for Duchenne Muscular Dystrophy

by Dr. Hsien-Hsien Lei
Posted October 18, 2007 in DNA and Disease, Gene Therapy

duchenneGene therapy corrects defective genes by inserting a normal one, repairing specific mutations, or regulating gene function. One new way specific genetic mutations can be targeted is by skipping them altogether. AVI BioPharma is developing exon skipping genetic technology, called ESPRIT (exon skipping pre–RNA interference technology), that can sidestep mutations that result in nonfunctioning or malfunctioning proteins. Duchenne muscular dystrophy (DMD) is the first genetic disorder in which ESPRIT will be tested.

Preclincial studies have shown that AVI’s NeuGene compounds can target the faulty exon, snip it out like fine genetic surgery, and force the cell machinery to skip over the fault and continue to splice and translate. The result is a form of dystrophin able to convert DMD to a form of the disease similar to the naturally–occurring and less severe BMD.

charley secklerEarlier this week, Charley’s Fund, Inc. awarded a $2.45 million research grant to AVI BioPharma to develop ESPRIT. Charley’s Fund is named for Charley Seckler who was diagnosed with DMD in July 2004. He is one of over 15,000 to 20,000 boys in the United States who have the genetic disorder. Current treatments include steroids and stretching but most DMD kids die in their late teens or early twenties.

Duchenne muscular dystrophy is caused by a mutation in the dystrophin gene on the X chromosome, one of the longest human genes. Because the gene is X-linked, boys are affected disproportionately; boys only have one X chromosome while girls have the protective effect of a normal dystrophin gene on the second of their X chromosomes. A normal dystrophin protein gives muscle cells strength and stability in addition to playing a role in carrying cell signals.

AVI has applied ESPRIT to diseases other than DMD. Exon skipping may also be a useful technique for treating type 1 diabetes, multiple sclerosis, and other inflammatory disorders.

Image: Duchenne muscule dystrophy deletion, FISH. Human chromosomes highlighted by fluorescent probes which bind to specific sequences of DNA. The FISH (fluorescence in situ hybridisation) study is of a female heterozygous for a deletion in the Duchenne muscular dystrophy (DMD) gene. The DMD cosmid probe is red, the X chromosome centromeres are green. via Wellcome images under Creative Commons.

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Gene Therapy Not Needed

by Dr. Hsien-Hsien Lei
Posted June 3, 2007 in DNA and Disease, Gene Therapy

The genome revolution isn’t all about re-engineering our genome. In a mis-titled article–Gene therapy: how science moved from hype to hope–Mark Henderson of The Times UK talks about the many different gene variants that have been identified recently that are common in the general population and yield a smaller effect compared to major gene mutations affecting only a small subset of the population, e.g., Huntington’s or cystic fibrosis. But rather than focusing on actually changing our genes, as the title would suggest, the article concludes with the idea that environmental factors are easier to manipulate than genes because gene variants with smaller effects are neither sufficient nor necessary for causing disease. I’m glad this point was emphasized so that people don’t fall into the trap of believing in genetic determinism.

As for the commenter who wondered why research focuses on disease-causing genes rather than disease-preventing, there’s an easy answer. Eliminating disease is what the medical model is based on. Perhaps the genome revolution will bring about another revolution in preventive medicine.
clipped from

Where genetics was once capable of pinning down only rare mutations with a catastrophic impact, it is now tracing variants with smaller effects that are much more widespread. You might call it the democratisation of the genome.

“What we are going to get out of this in the end is not genetic engineering, but environmental engineering,” Dr Plomin said. “A lot is going to be about changing behaviour, about education. It’s not always going to be: you’ve got this genetic problem and here’s a pill. Most genetic effects are going to be too small for that.”

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Zorro Locked Nucleic Acid Silences Genes

by Dr. Hsien-Hsien Lei
Posted May 2, 2007 in Gene Therapy

zorro posterMutated genes beware. Zorro LNA (locked nucleic acid) is in town.

Researchers at the Karolinska Institute in Stockholm, Sweden have developed a method of shutting down mutant genes using Zorro-LNA (pdf), a synthetic analog of nucleic acids that can bind to both strands of a gene’s DNA and silence it. Potential uses for Zorro-LNA may be to deactivate genes that cause severe diseases such as Huntington’s disease, familial high cholesterol, polycystic kidney disease, some instances of glaucoma, colorectal cancer, and neurofibromatosis.

Dr. Gerald Weissmann, editor-in-chief of The Faseb Journal is very optimistic:

This is a major development in the treatment not only of genetic diseases, but also of acquired diseases when microbes or toxins cause genes to go awry. One might say these researchers have found a gene-hunter’s Holy Grail for which scientists have been hunting for many years. Zorro-LNA should give us a new, safe way of blocking the effects of errors in our genetic repertoire.

Based on the paper’s abstract, Zorro locked nucleic acid induces sequence-specific gene silencing, it appears the results are limited to in vitro experiments. Cool substance, cool name, cool idea, but perhaps a little too early to be invoking religious references?

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Eye on DNA, Literally

by Dr. Hsien-Hsien Lei
Posted May 1, 2007 in DNA and Disease, Gene Therapy

blue eyeRobert Johnson should be Eye on DNA’s spokesperson. The 23-year-old is the first recipient of gene therapy to correct a faulty copy of the RPE65 gene that causes severe retinal degenerative disease. The surgery involved injecting functional copies of the RPE65 gene mixed with a viral vector into the back of the eye underneath the retina. Several years ago, gene therapy successfully restored the vision of dogs with inherited retinal degerative disease.

The RPE65 gene on chromosome 1 encodes a protein that is found in the the retinal pigment epithelium. The protein is involved in allowing specific compounds to enter the visual cycle for processing. Other forms of RPE65 also help to move vitamin A that plays a part in nourishing the retina. A number of disorders are affected by RPE65 genetic mutations including Leber’s congenital amaurosis and juvenile retinitis pigmentosa, both are inherited.

Professor Robin Ali of the UCL Institute of Ophthalmology:

There are many forms of retinal degeneration, meaning the use of gene therapy treatments must be individually developed then tested in a separate clinical trial specifically for that disease. However, the results from this first human trial are likely to provide an important basis for many more gene therapy protocols in the future, as well as potentially leading to an effective treatment for a rare but debilitating disease.

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