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Drug-Based Therapy News

Nikolas L. Jorstad, Matthew S. Wilken, William N. Grimes, Stefanie G. Wohl, Leah S. VandenBosch, Takeshi Yoshimatsu, Rachel O. Wong, Fred Rieke, & Thomas A. Reh

NEI-funded researchers use a clue from zebrafish to discover the cues that reprogram Müller glia into retinal neurons.

Sarath Vijayakumar Frederic F. Depreux Francine M. Jodelka Jennifer J. Lentz Frank Rigo Timothy A. Jones Michelle L. Hastings.

These findings provide the first direct evidence of an effective treatment of peripheral vestibular function in a mouse model of USH1C and reveal the potential for using antisense technology to treat vestibular dysfunction.

Harvard Stem Cell Institute (HSCI) researchers at Brigham and Women’s Hospital (BWH) and Massachusetts Eye and Ear Infirmary and colleagues from Massachusetts Institute of Technology (MIT) have developed an approach to replace damaged sound-sensing hair cells, which eventually may lead to therapies for people who live with disabling hearing loss.

Researchers are working to find how fish can regenerate their eyes after they have been injured and if there is a way to make this happen the same way in a human eye.

Scientists from Brigham and Women’s Hospital (BWH), MIT, and Massachusetts Eye and Ear believe they may have found a way to treat hearing loss by regenerating hair cells in the inner ear and hope to begin clinical trials in 18 months.

Frederic F. Depreux, Lingyan Wang, Han Jiang, Francine M. Jodelka, Robert F. Rosencrans, Frank Rigo, Jennifer J. Lentz, John V. Brigande and Michelle L. Hastings.

ASO delivery to the intra-amniotic cavity modulates neonatal gene expression and may serve as a therapeutic intervention in itself or when paired with a suitable postnatal therapeutic strategy. Further optimization of the method will broaden the potential impact and applicability of this approach.

Kumar N Alagramam, Suhasini R Gopal, Ruishuang Geng, Daniel H-C Chen, Ina Nemet, Richard Lee, Guilian Tian, Masaru Miyagi, Karine F Malagu, Christopher J Lock, William R K Esmieu, Andrew P Owens, Nicola A Lindsay, Krista Ouwehand, Faywell Albertus, David F Fischer, Roland W Bürli, Angus M MacLeod, William E Harte, Krzysztof Palczewski & Yoshikazu Imanishi.

A new study published in Nature Chemical Biology reports the first small molecule targeted therapy for progressive hearing loss in a mouse model of USH3, an USH classified by progressive loss of hearing and vision starting in the first few decades of life along with variable balance disorder.

Rajarshi Ghosh, Wang Likun, Eric S. Wang, B. Gayani K. Perera, Aeid Igbaria, Shuhei Morita, Kris Prado, Maike Thamsen, Deborah Caswell, Hector Macias, Kurt F. Weiberth, Micah J. Gliedt, Marcel V. Alavi, Sanjay B. Hari, Arinjay K. Mitra, Barun Bhhatarai, Stephan C. Schürer, Erik L. Snapp, Douglas B. Gould, Michael S. German, Bradley J. Backes, Dustin J. Maly, Scott A. Oakes, and Feroz R. Papa.

Allosteric inhibition of the IRE1α RNase preserves cell viability and function during endoplasmic reticulum stress.
New research shows that modifying a particular protein, IRE1, can put off cell death.

Jennifer J Lentz, Francine M Jodelka, Anthony J Hinrich, Kate E McCaffrey, Hamilton E Farris, Matthew J Spalitta, Nicolas G Bazan, Dominik M Duelli, Frank Rigo & Michelle L Hastings

New research shows that hearing and vestibular function can be rescued in a mouse model of Usher 1c using an antisense oligonucleotide.

"Ray of hope for human Usher syndrome patients": Uwe Wolfrum and his colleagues at Johannes Gutenberg University Mainz are increasing our understanding of Usher syndrome.

BioDiem has strengthened the preclinical case for its BDM-E eye disease drug with further positive results from formal studies that will help progress out-licensing opportunities for the drug. BDM-E has received Orphan Drug designation from the United States Food and Drug Administration for the treatment of the inherited degenerative eye disorder, retinitis pigmentosa.

This study, conducted in mice modelling the human disease retinitis pigmentosa, showed that the drug norgestrel could "rescue" light-detecting retinal cells. The synthetic progestin hormone, an active component of the contraceptive "mini-Pill," allowed mice which should have gone blind to retain their sight. A new study is now planned for next year to see if humans experience the same protective effects.

New treatment for nonsense mutations may soon be ready for use in Usher syndrome patients. A molecule known as PTC124 appears to cause the stop signal in a mutated USH1C to be ignored, allowing the protein to be formed normally in cell cultures.

Neurotech announced in Investigative Ophthalmology and Visual Science that their NT-501 implant slowed the loss of photoreceptors in three patients, including one with Usher syndrome type 2.

Jennifer Phillips, Ph.D., reviewed and put together a 'FAQ' on a small observational study of the effects of Valproic Acid, which was published in the summer of 2010 online in the British Journal of Ophthalmology.

QLT091001 is an orally administered synthetic retinoid replacement for 11-cis-retinal, which is a key biochemical component of the visual retinoid cycle, and is under investigation for the treatment of LCA and RP.

Some unexpected effects of lead exposure that may one day help prevent and reverse blindness have been uncovered.

Researchers at Trinity College Dublin have reported the development of a new drug delivery system which has the potential to treat degenerative diseases of the retina, including retinitis pigmentosa.

Neurotech Pharmaceuticals, Inc., today announced that the Company's product candidate, NT-501 demonstrated a strong biologic effect in two Phase 2 clinical trials for retinitis pigmentosa (RP)

Aminoglycocides have shown promising effects in cell cultures and may someday be used to suppress mutations involved in Usher syndrome.

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