Archive by tag | gene therapy

As gene therapy technologies blossom, ddRNAi tries to take root

Posted by Nicholette Zeliadt | Categories:

As gene therapy technologies blossom, ddRNAi tries to take root

Before there was Twitter, there was Facebook, and before that, Friendster. And who can forget MySpace? There’s a similar trend of successive usurping technologies in the fast-moving quest to develop therapeutics capable of modifying the genome. Since the late nineties, we’ve witnessed the rise of several gene-silencing approaches, from “antisense” oligonucleotides and RNA interference (RNAi) to the latest targeted genome-editing techniques, such as those based on zinc finger nucleases or CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) technology. These rapid developments raise the stakes for companies that have wagered on a particular gene-silencing approach.  Read more

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Gene therapy to restore hearing sounds closer to reality after success in deaf-born mice

Posted by Kathleen Raven | Categories:

Gene therapy to restore hearing sounds closer to reality after success in deaf-born mice

Using gene therapy, a team of researchers for the first time successfully restored normal hearing to mice born deaf due to a missing protein, according to a study published today in the journal Neuron. This finding could be music to the ears of people whose congenital hearing loss is caused by genetic mutations that may prevent tiny inner ear hairs from interacting with neurotransmitters that are necessary for hearing. In the current experiment, mice recovered full hearing for an average of seven weeks, with two of 19 mice maintaining it for as long as one and a half years. “I was completely shocked,” says lead author Lawrence Lustig, director of the Douglas Grant Cochlear Implant Center at the University of California, San Francisco. “The hearing looked almost completely normal and you couldn’t tell these were rescued mice.”  … Read more

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