University of Missouri-Columbia · Microbiology & ImmunologyDuan Gene Therapy Lab
Dongsheng Duan · Columbia, Missouri
Research Focus
Dr. Dongsheng Duan's research lab focuses on developing innovative gene therapies for Duchenne muscular dystrophy (DMD), a severe muscle-wasting condition. The lab employs advanced techniques like adeno-associated virus (AAV) delivery systems and CRISPR gene editing to enhance treatment efficacy and safety. By utilizing models, especially canines that closely mimic human DMD, they aim to refine therapeutic approaches and address challenges such as immune responses and treatment longevity.
Duchenne muscular dystrophygene therapyAAVCRISPRmuscle deliverycanine modeltherapeutic editingmicrodystrophintoxicity studyimmune response
Research Aims
5AIM 01Develop effective AAV Mediated gene therapies for Duchenne muscular dystrophy.
AIM 02Explore novel strategies to overcome Cas9-induced immune responses.
AIM 03Identify optimal AAV capsids for systemic delivery in human muscle.
AIM 04Assess long-term therapeutic benefits in canine DMD models.
AIM 05Reduce the toxicity associated with high-dose AAV administration.
Skills the Lab Values
AAV vector designCRISPR/Cas9 gene editingmouse and canine model experimentssystemic delivery methodscell culture techniquesgene knock-in/out strategiesmuscle physiology assessmentshistological analysisprotein expression analysisxenograft procedures
Output against the department
Publications302top 16% of dept.78th of 486
this lab
most publishedleast published
Citations16ktop 24% of dept.116th of 486
this lab
most citedleast cited
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