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Drug Design Lab

Virtual Drug Design

Drug design refers to the inventive process of creating new compounds and medications based on our knowledge of a biological target. Often, this biological target is a protein that may be inhibited to provide a therapeutic effect. A detailed understanding of a protein’s structure and function is often required in the design of novel inhibitors. The Mahajan laboratory utilizes virtual, or computer-aided, drug design methods to model new compounds that are complementary to a target protein’s shape and charge. These virtually-designed lead-like compounds are then synthesized in collaboration with Stanford’s Medicinal Chemistry Knowledge Center (MCKC) for biochemical and cellular testing.


Drug Repositioning

The development of new drugs can cost upward of a billion dollars and take over a decade before the drug reaches the market. Drug repositioning makes use of existing drugs for the treatment of diseases where there are few therapeutic options. This approach can provide a safer alternative to the development of new compounds, since repurposed drugs are already FDA-approved, have proven bio-availabilities, and well-characterized side-effect profiles. To identify which drugs to reposition, our laboratory performs proteomic analyses of liquid biopsies (e.g. vitreous or aqueous humor) from diseased patients to identify drug targets and biomarkers. This approach allows for rapid, real-time repositioning of available drugs to patients with few therapeutic options. 


Structure-based drug design for inherited eye diseases.
Identifying available drugs to treat patients with rare diseases using proteomics.


Jul 16 2020 | Posted In: 20/20 Blog
Apr 29 2020 | Posted In: 20/20 Blog
Palo Alto, CA — Traumatic brain injuries (TBI) can happen when someone is close to an explosion that sends out powerful shock waves through the air.
Apr 20 2020 | Posted In: 20/20 Blog
Palo Alto, CA — Is there something to feed retinal cells that can give them the energy to withstand gene mutations that make them sick?
Mar 3 2020 | Posted In: 20/20 Blog
Dylan Parsons, a postdoctoral fellow in the Mahajan Lab, was one of three candidates selected to receive an award from the Stanford T32 Vision Training Grant. The grant provides one year of funding for intensive bench to bedside translational research aimed at bringing new therapies to patients.
Dec 4 2019 | Posted In: 20/20 Blog
Palo Alto, CA — Research from the Mahajan lab was featured on the cover of Human Mutation'sDecember 2019 issue, highlighting their manuscript, “CAPN5 genetic inactivation phenotype supports therapeutic inhibition trials.” 


Translation of CRISPR Genome Surgery to the Bedside for Retinal Diseases., Xu, Christine L., Cho Galaxy Y., Sengillo Jesse D., Park Karen S., Mahajan Vinit B., and Tsang Stephen H. , Front Cell Dev Biol, 2018, Volume 6, p.46, (2018)
Bevacizumab injection in patients with neovascular age-related macular degeneration increases angiogenic biomarkers, Cabral, Thiago, Lima Luiz H., Mello Luiz Guilherme, Polido Júlia, Correa Éverton P., Oshima Akiyoshi, Duong Jimmy, Serracarbassa Pedro, Regatieri Caio V., Mahajan Vinit B., et al. , Ophthalmology Retina, Volume 2, p.31–37, (2018)
Personalized proteomics in proliferative vitreoretinopathy implicate hematopoietic cell recruitment and mTOR as a therapeutic target, C Roybal, Nathaniel, Velez Gabriel, Toral Marcus A., Tsang Stephen H., Bassuk Alexander G., and Mahajan Vinit B. , American journal of ophthalmology, (2017)
Retinal and choroidal angiogenesis: a review of new targets, Cabral, Thiago, Mello Luiz Guilherme, Lima Luiz H., Polido Júlia, Regatieri Caio V., Belfort Rubens, and Mahajan Vinit B. , International journal of retina and vitreous, Volume 3, Number 1, p.31, (2017)
Therapeutic drug repositioning using personalized proteomics of liquid biopsies, Velez, Gabriel, Bassuk Alexander G., Colgan Diana, Tsang Stephen H., and Mahajan Vinit B. , JCI insight, Volume 2, (2017)
Ocular hypertension after intravitreal dexamethasone (Ozurdex) sustained-release implant, Chin, Eric K., Almeida David R. P., Velez Gabriel, Xu Kunyong, Peraire Maria, Corbella Maria, Elshatory Yasser M., Kwon Young H., Gehrs Karen M., H Boldt Culver, et al. , Retina, Volume 37, p.1345–1351, (2017)