Down syndrome (DS) is characterized by specific learning/memory (L/M) deficits and brain abnormalities. Many of these features develop after birth, suggesting the possibility of effective postnatal therapeutics to prevent their onset or reduce their severity. This goal is supported by recent successes, with several pharmacological treatments, in rescuing L/M deficits in the Ts65Dn mouse model of DS. Extending these observations to safe, effective human clinical trials requires assimilation of functional informatin on chromosome 21genes, detailed understanding of the DS cognitive phenotype, use of appropriate model systems to test efficacy of drugs prior to human trials, and exploration of a broad range of pharmacological regimens in order to identify those of the greatest potential. No conference focusing on basic, clinical and translational research for intellectual disability in DS has been held in approximately 5 years. This application requests funding to cover expenses for a conference to bring together investigators from these diverse research areas. Participants will include established DS and non-DS investigators whose research directly or indirectly informs aspects of the DS cognitive phenotype and/or responses of these features to pharmacological therapy. The meeting will be held for three days in Washington DC in April 2013. It is anticipated that this conference will lead to new hypotheses regarding pharmacological treatment of cognitive deficits in DS, foster new collaborations and bring new researchers with novel expertise into the field of DS.
Down syndrome (DS) is characterized by specific learning/memory (L/M) deficits and brain abnormalities. Many of these features develop after birth, suggesting the possibility of effective postnatal therapeutics to prevent their onset or reduce their severity. This goal is supported by recent successes, with several drug treatments, in rescuing L/M deficits in one mouse model of DS. Extending these observations to safe, effective human clinical trials requires integrating information obtained from areas of basic, clinical and translational research that are relevant to intellectual disability. This application requests funding to cover expenses for a conference to bring together investigators from these diverse research areas. Participants will include established DS and non-DS investigators whose research directly or indirectly informs aspects of intellectual disability in DS and/or responses of these features to pharmacological therapy. The meeting will be held for three days in Washington DC in April 2013. No conference on this topic has been held within the last five years and it is anticipated that this will lead to new hypotheses regarding pharmacological treatment of cognitive deficits in DS, foster new collaborations and bring new researchers with novel expertise into the field of DS.
| Busciglio, J; Capone, G; O'Bryan, J et al. (2013) Down syndrome: genes, model systems, and progress towards pharmacotherapies and clinical trials for cognitive deficits. Cytogenet Genome Res 141:260-71 |
