1. Modeling Gene Therapy for Intractable Developmental and Epileptic Encephalopathy Aimiuwu, Osasumwen Virginia 2021 Theses BiologyEpilepsy--Genetic aspectsMutagenesisBrain--Diseases--TreatmentMice--GeneticsGene therapy
2. Engineering a versatile lipoMSN delivery platform for the development of gene and drug therapies Gong, Jing 2020 Theses NanotechnologyDrugsGene therapyChemotherapyCRISPR (Genetics)Nanoparticles
3. Correction of Monogenic and Common Retinal Disorders with Gene Therapy Sengillo, Jesse D.; Justus, Sally; Silva, Thiago George Cabral; Tsang, Stephen H. 2017 Articles Monogenism and polygenismRetina--Diseases--TreatmentGene therapyBiochemistry
4. The Development of an Inducible Akt as a Potential Gene Therapy for Parkinson’s Disease Park, Soyeon 2017 Theses BiologyNeurosciencesParkinson's diseaseGene therapyProtein kinases
5. Restriction of HIV-1-based lentiviral vectors in adult primary marrow-derived and peripheral mobilized human CD34+ hematopoietic stem and progenitor cells occurs prior to viral DNA integration Griffin, Daniel; Goff, Stephen P. 2016 Articles Gene therapyLentivirusesStem cellsHIV infectionsMolecular biology
6. Patient-Specific iPSC-Derived RPE for Modeling of Retinal Diseases Nguyen, Huy V.; Li, Yao; Tsang, Stephen H. 2015 Articles Retina--DiseasesOphthalmologyGene therapyRetina--Diseases--TreatmentRetinal degeneration--Alternative treatmentRetinitis pigmentosa