{"doi":"10.3410/f.739808595.793584193","title":"Faculty Opinions recommendation of Presymptomatic training mitigates functional deficits in a mouse model of Rett syndrome.","abstract":"Mutations in the X-linked gene MECP2 cause Rett syndrome, a progressive neurological disorder in which children develop normally for the first one or two years of life before experiencing profound motor and cognitive decline 1,2,3 . Currently, there are no effective treatments for Rett syndrome, but we hypothesized that using the period of normal development to strengthen motor and memory skills might confer some benefit. We found that intensive training beginning in the presymptomatic period dramatically improved the performance of specific motor and memory tasks in a mouse model of Rett syndrome and significantly delayed the onset of symptoms. These benefits were not observed when the training was initiated only after symptom onset. Markers of neuronal activity and chemogenetic manipulation revealed that task-specific neurons that are repeatedly activated during training develop more dendritic arbors and have better neurophysiological responses, thereby enhancing their functionality and delaying symptom onset. These results provide a rationale for newborn genetic screening for Rett syndrome, as they suggest that presymptomatic intervention might mitigate symptoms or delay their onset. Similar strategies should be studied for other childhood neurological disorders.","journal":"Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature","year":2021,"id":229651,"datarank":0.0,"base_score":0.0,"endowment":0.0,"self_citation_contribution":0.0,"citation_network_contribution":0.0,"self_endowment_contribution":0.0,"citer_contribution":0.0,"corpus_percentile":null,"corpus_rank":null,"citation_count":0,"citer_count":0,"citers_with_citation_signal":0,"citers_with_endowment":0,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.9555,"is_data_producer":false,"deposit_databanks":null,"is_oa":true,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":"2021-01-01","fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":327116,"name":"Santhosh Girirajan","orcid":"0000-0003-0598-4945","position":0,"is_corresponding":true}],"reference_count":43,"raw_metadata":null,"created_at":"2026-07-18T23:55:01.675482Z","pmid":null,"pmcid":null,"fwci":null,"citation_percentile":null,"influential_citations":0,"oa_status":null,"license":null,"views":0,"total_file_size_bytes":0,"version_count":0,"fair_f":null,"fair_a":null,"fair_i":null,"fair_r":null,"fair_zscore":null,"fair_rationale":null,"fair_model":null,"fair_agent_version":null,"fair_fulltext_source":null,"fair_has_llm":null,"fair_computed_at":null,"clinical_trials":[],"software_tools":[],"db_accessions":[],"linked_datasets":[],"topics":[]}