rAAVrh74.MHCK7.DYSF.DV for Treatment of Dysferlinopathies
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02710500|
Recruitment Status : Active, not recruiting
First Posted : March 16, 2016
Last Update Posted : May 13, 2019
Nationwide Children's Hospital
Information provided by (Responsible Party):
Jerry R. Mendell, Nationwide Children's Hospital
No Study Results Posted on ClinicalTrials.gov for this Study
|Recruitment Status :||Active, not recruiting|
|Estimated Primary Completion Date :||July 2019|
|Estimated Study Completion Date :||July 2019|
Sondergaard PC, Griffin DA, Pozsgai ER, Johnson RW, Grose WE, Heller KN, Shontz KM, Montgomery CL, Liu J, Clark KR, Sahenk Z, Mendell JR, Rodino-Klapac LR. AAV.Dysferlin Overlap Vectors Restore Function in Dysferlinopathy Animal Models. Ann Clin Transl Neurol. 2015 Mar;2(3):256-70. doi: 10.1002/acn3.172. Epub 2015 Jan 20.
Grose WE, Clark KR, Griffin D, Malik V, Shontz KM, Montgomery CL, Lewis S, Brown RH Jr, Janssen PM, Mendell JR, Rodino-Klapac LR. Homologous recombination mediates functional recovery of dysferlin deficiency following AAV5 gene transfer. PLoS One. 2012;7(6):e39233. doi: 10.1371/journal.pone.0039233. Epub 2012 Jun 15.