This week, we have two top-tier guests explaining some pretty remarkable breakthroughs published this summer in their respective fields of MS and dermatology, respectively.
Sergio Baranzini, PhD (UCSF), discusses recently published research on a genetic breakthrough in understanding MS progression. Dr. Baranzini’s research involves large analyses of samples from patients with MS to characterize the activity of genes during 1) different stages of the disease (eg, remission vs. relapse), 2) differential response to treatment (eg, good responders vs. poor responders), and 3) disease progression (eg, benign vs. severe).
Also, Peter Marinkovich, MD (Stanford) explains a genetic breakthrough in the treatment of the severe skin condition recessive dystrophic epidermolysis bullosa. We learn about this condition and how Dr. Marinkovich’s team has developed an effective gene therapy that has shown very promising results in their recently published clinical trial.
Enjoy Listening!
Additional reading
International Multiple Sclerosis Genetics Consortium; MultipleMS Consortium. Locus for severity implicates CNS resilience in progression of multiple sclerosis. Nature. 2023 Jul;619(7969):323-331
Gurevich I, Agarwal P, Zhang P, Dolorito JA, Oliver S, Liu H, Reitze N, Sarma N, Bagci IS, Sridhar K, Kakarla V, Yenamandra VK, O'Malley M, Prisco M, Tufa SF, Keene DR, South AP, Krishnan SM, Marinkovich MP. In vivo topical gene therapy for recessive dystrophic epidermolysis bullosa: a phase 1 and 2 trial. Nat Med. 2022 Apr;28(4):780-788.
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