RNA and oligonucleotide therapies are in the vanguard of scientific research making the transition from the bench to the clinic. These approaches will in time lead to better and more targeted therapies against major neurological diseases, including Duchenne muscular dystrophy, spinal muscular atrophy, amyotrophic lateral sclerosis, frontotemporal dementia, familial dysautonomia and myotonic dystrophy. Many of these technologies are of relevance to other diseases, and we have appended a list of other institutes that may be interested in providing partial support. Tremendous value is gained by bringing together groups and individuals who are working with different technologies in a variety of models of human disease. Few academic meetings cover the focused ground of this meeting, with a specific emphasis on RNA and oligonucleotide therapies. The meeting will bring together cutting-edge basic, translational, and clinical research, and will attract specialists from industr as well as from academia, ensuring a """"""""real-world"""""""" perspective on these important challenges. The narrow emphasis is a strong plus of the Cold Spring Harbor style meeting, driven as much by the openly submitted abstracts as by the invited speakers. The proposed program will include keynote speakers, invited speakers and short talks selected from openly submitted abstracts. Major themes include: Exon Skipping Therapy;siRNA &miRNA;Aptamers as Therapeutics;Oligonucleotide Therapy;Delivery of RNA Therapeutics;and Long Non-Coding RNAs. Because the program is assembled in part from openly submitted abstracts, the final program is not decided until a couple of months before the meeting, ensuring that the latest advances and discoveries can be included in the meeting. Two of the three organizers are active in the development of novel therapies specifically targeted towards neurological disorders, and so we anticipate a significant proportion of presented science to be relevant to the treatment of neurological and neuromuscular diseases. Participants will be drawn from academic labs, research institutes and biotech/pharmaceutical industries, and will include leaders in the field, established investigators, junior faculty, postdoctoral fellows, graduate students and corporate scientists. Special effort will be made to encourage participation by underrepresented constituencies and next generation scientists in the program. Each session will be chaired by a leading scientist in the field selected by the organizers. Of special importance are the poster sessions, where many participants can present their work in an atmosphere conducive to informal discussion. The meeting will be of moderate size and we expect about 150-200 people to attend, the vast majority of whom will be presenting a poster or talk.

Public Health Relevance

The primary repository for genetic information lies in our DNA, present in almost all the cells in our bodies. RNA, long understood to be the intermediary between our genes and the proteins they encode, has in recent years been shown to play an even more central role in our cells, with recent discoveries of a host of novel processes by which small RNAs regulate gene expression. In turn this has led to the development of new concepts for human diseases, including a variety of devastating neurological disorders such as Duchenne muscular dystrophy and amyotrophic lateral sclerosis. Through a combination of novel targets, strategies and chemistries, scientists involved in drug design using RNA and related oligonucleotides - short nucleic acids and related compounds - now have an arsenal of approaches to explore and develop. The proposed CSHL meeting will bring together world leaders in the above-mentioned disciplines in a medium-size meeting format at a location that has a history of running outstanding conferences. In doing so, the meeting is likely to foster interactions that will lead to discoverin new ways of controlling many neurological disorders and other diseases.

National Institute of Health (NIH)
National Institute of Neurological Disorders and Stroke (NINDS)
Conference (R13)
Project #
Application #
Study Section
National Institute of Neurological Disorders and Stroke Initial Review Group (NSD)
Program Officer
Porter, John D
Project Start
Project End
Budget Start
Budget End
Support Year
Fiscal Year
Total Cost
Indirect Cost
Cold Spring Harbor Laboratory
Cold Spring Harbor
United States
Zip Code