University of Missouri-Columbia · BiologyLorson Gene Therapy Lab
Christian L. Lorson · Columbia, Missouri
Research Focus
Dr. Christian L. Lorson's lab focuses on developing gene therapies for inherited neurological disorders, particularly Charcot-Marie-Tooth disease and related conditions. The lab works on creating animal models that mimic human diseases to understand the biological processes involved and to test potential therapeutic approaches. Ultimately, the goal is to develop effective treatments that can improve the quality of life for affected individuals.
Charcot-Marie-Tooth Diseasegene therapyneurodegenerationmouse modelsIGHMBP2 mutationsneurological disordersSMA with Respiratory Distresspre-rRNA processingneurofilamenttherapeutic development
Research Aims
5AIM 01Develop a precision medicine-based therapeutic for CMT2E using a novel mouse model.
AIM 02Investigate how Ighmbp2 mutations contribute to neurodegenerative diseases.
AIM 03Understand the functional consequences of specific Ighmbp2 mutations on disease progression.
AIM 04Assess the therapeutic potential of novel gene delivery systems for CMT.
AIM 05Explore the relationship between IGHMBP2 mutations and pre-rRNA processing.
Skills the Lab Values
animal model developmentgene therapy techniquesbiochemical assaysmolecular biology techniquesPCRRNA processingmouse geneticsprotein expression analysistherapeutic vector design
Output against the department
Publications156top 43% of dept.444th of 1,033
this lab
most publishedleast published
Citations9.5ktop 40% of dept.415th of 1,033
this lab
most citedleast cited
Career totals from OpenAlex, compared against labs that share a department. A department spans everything from one paper to tens of thousands, so bar heights are on a log scale — otherwise the largest lab would flatten every other bar to a line.