Gorica Margulis

I’m thrilled to share that our company has successfully launched the Iranian version of Risdiplam into the domestic market. This groundbreaking drug is designed for the treatment of spinal muscular atrophy (SMA), a rare genetic disorder that primarily affects motor neurons and muscle function. Although my role in this project was very negligible, I am proud to have been part of a dedicated team that turned this vision into reality. This accomplishment demonstrates the incredible power of collaboration and innovation in the pharmaceutical field. Even the smallest contributions can lead to meaningful change. Risdiplam is an oral SMN2 splicing modifier that works by increasing the production of the survival motor neuron (SMN) protein, which is deficient in SMA patients. By stabilizing RNA splicing of the SMN2 gene, it ensures the production of functional SMN protein critical for motor neuron survival. This drug was approved by the U.S. FDA in 2020 under the name Evrysdi and has been recognized for its efficacy in all SMA types. Its approval was based on clinical trials like FIREFISH and SUNFISH, which demonstrated significant motor function improvements and quality of life enhancements in SMA patients. Our team’s effort in developing a local version of this life-changing therapy is a huge step forward for patients in need. #Risdiplam #SMA #PharmaceuticalInnovation #Ronakpharmaceuticalco #Teamwork #ProudMoment #Ronagen #everligen

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What separates ResolveDNA WGA from other whole genome amplification kits out there? Hear from Aleksei Mikhalchenko, PhD, Genome Informatics Lead at the Center for Embryonic Cell and Gene Therapy, Oregon Health & Science University ⤵   "Our research depends critically on the ability to sequence single-cell DNA and accurately identify its genomic variants. Among all the whole-genome amplification (WGA) methods we have tested, PTA-based WGA has provided the most accurate output. It reliably stays close to the original 50/50 ratio of allele frequency in heterozygous loci, minimizing allele dropout and false-positive loss of heterozygosity. This makes it an invaluable tool for our research." Don’t settle for whole genome amplification kits that make you choose between high fidelity, even, or complete coverage. Our novel PTA-powered ResolveDNA WGA kits provide all three. Learn more: https://lnkd.in/gm37WwEJ

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Really interesting approach to ADC therapeutics with conjugation to silencing mRNA.

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💎💎💎Whatsapp!! 💎💎💎Engineered Human Plasma B Cells Secrete Bispecifics to Treat Leukemia in Mice Researchers headed by a team at Seattle Children’s Research Institute have shown for the first time that #engineered #human #plasma B cells can be used to treat a #disease—more specifically #leukemia—in a #humanized #mouse model. The results mark a key step in the realization of engineered plasma cells (ePCs) as #therapies to treat #cancer, #autoimmune disorders, and #protein deficiency disorders. “We hope that this proof-of-concept study is the first of many applications of engineered plasma B cells, and eventually will lead to a single-shot therapeutic,” said Richard James, PhD, at the Seattle Children’s Research Institute. “Because engineered plasma B cells can live for a very long time, greater than 10 years, they could be used as a long-term source of many #biologic #drugs.” James is the senior author of the team’s published paper in Molecular Therapy, titled “Human plasma cells engineered to secrete bispecifics drive effective in vivo leukemia killing,” in which they stated, “These findings support further development of ePCs for use as a durable delivery system for the treatment of #acute #leukemias, and #potentially other cancers.” ➡ 💎 You can find more pieces of work by clicking here. https://lnkd.in/eSG67K5G https://lnkd.in/dB3s88ei #Engineered_Human #Plasma_B_Cells #Leukemia #Mice #engineered #human #plasma #disease—more specifically #leukemia #therapies #cancer, #autoimmune #protein

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My initial research focused on the impact of pollutants on the genomes of marine organisms😅, presented at the 7th National Conference on Innovation and Technology in Biological Sciences and Chemistry of Iran. This study examined marine pollutants and their effects on individual organisms, highlighting the troubling reality that our lives are filled with pollution💔, jeopardizing smaller life forms. https://lnkd.in/ghFvUT_T #biotechnology #biology

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Excited to share our work, just published in Nature communications, on elucidation of the cooperative DNA binding mechanism of master transcription factor and cancer driver ONECUT2. The CUT and homeodomain are ubiquitous DNA binding motifs that are found paired in multiple transcription factor families in humans, including ONECUT, POU, SATB and CUX. The CUT-homeodomain group of transcription factors are key determinants for cell differentiation, and organismal development, with implications in disease. We carried out an integrative analysis showing the homeodomain of ONECUT2 drives its energetically favorable interaction to a cognate promoter DNA, by allosterically inducing a conformational rearrangement in CUT. Zooming in, the favorable energetics is dependent on evolutionarily conserved DNA base-specific interactions by both the CUT and homeodomain. We also identify an arginine pair in the homeodomain of ONECUT2 that interacts uniquely to the DNA compared to the other families listed above. Notably, these interactions are necessary for the OC2-driven transcriptional program in a cell-based prostate cancer model. Overall, this work brings forth an elegant crosstalk between the CUT and homeodomain, that determines their cooperative DNA binding mechanism. This is the result of an outstanding teamwork and highlights the importance of curiosity-driven research in the development of potential therapeutic strategies. Read the article here: https://lnkd.in/gaNYkCcB

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🎙️ New episode on Getting to Know Your Research! 🎉 In episode seven, Dr. Dawn Eichenfield and Dr. Jennifer Sui discuss their 2022 PeDRA Research Fellowship project titled Characterization of non-coding genetic loci and transcription factors that drive the development of linear morphea. Listen to learn about the many collaborations needed to propel a study like this. 🎧 Listen on PeDRA Pearls or click here 👉 https://lnkd.in/eG5Atjeg #PeDRA #pedraresearch #dermatology #pedsderm #dermresearch #dermnerds #academicdermatology #dermpodcast #morphea

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There are some diseases that just don't have animal models; #Crohn's disease (CD) is one of them. 🎗 Kudos to UC San Diego UC San Diego HUMANOID: Human Organoid Center of Research Excellence (CoRE) for building a living biobank of human preclinical models for studying CD (adult stem cell derived #organoids) that are #benchmarked for #model #accuracy through rigorous computational metrics. The team discovered that despite the thunderous heterogeneity in clinical subtypes, all patients fall into just two major #disease-driving intrinsic #epithelial #phenotypes at the molecular level. Each requires its own personalized therapeutic fix with no crossover benefits! Read more (👇) on how a project this large in scope could only be pulled off because of team effort spanning the #clinic, the #bench, and the #cloud. Most importantly, HUMANOID's models are built in close collaboration with with clinician-scientists who command domain expertise; in this instance, we are grateful to UC San Diego IBD Clinic Directors, William Sandborn and Brigid Boland for access to their insights, expertise, patients, and clinics. Link to news: https://lnkd.in/gb2JTqG2   Link to article: https://lnkd.in/gZFUuF9Z A big shout out to the team of co-authors: Courtney Tindle, Ayden Fonseca, @sahar Taheri, Gajanan Dattatray Katkar, Jasper Lee, Priti Prasanna Maity, Ibrahim M Sayed, Stella-Rita Ibeawuchi, Eleadah Vidales, Rama Pranadinata, Mackenzie Fuller, Dominik Stec, Mahitha Shree Anandachar, Kevin Perry, @helen N. Le, Jason Ear, Brigid Boland, William Sandborn, Debashis Sahoo, Soumita Das and Pradipta Ghosh. #Grateful to #funders: The Helmsley Charitable Trust, and in part by National Institute of Allergy and Infectious Diseases (NIAID), National Center for Advancing Translational Sciences (NCATS).

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After 20+ episodes of written content, it is time for a change. So Manel and I decided to start also the “ Gene Editing in 180 seconds” series where we jump straight to the point covering some of the newest innovations in the genome editing field! In this one we tell you about eePASSIGE. Which built on the Prime Editig approach, coupled with an evolved recombines to integrate large cargos! Happy to receive your feedback, we hope you enjoy and we will see you in the next one! #crispr #wdc #cmn #genomediting

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🔬 Breakthrough in Cancer Research 🔬 UC Irvine's team led by Remi Buisson has identified two key enzymes, APOBEC3A and APOBEC3B, as new targets for cancer treatment strategies. This landmark discovery highlights their role in cancer mutation, disease progression, and resistance to treatments. Dr. Buisson’s work was initially funded by the UCI Anti-Cancer Challenge in 2018. Support from the UCI Anti-Cancer Challenge propels essential research efforts like these, moving us closer to defeating cancer. Read more about how this research could transform cancer therapy at https://lnkd.in/gbkRC3aU. #cancerresearch #cancertreatment #supportcancerresearch

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Varveri, A., Papadopoulou, M., Papadovasilakis, Z. et al. Immunological synapse formation between T regulatory cells and cancer-associated fibroblasts promotes tumour development. Nat Commun 15, 4988 (2024). https://lnkd.in/dZYrmzCd "Abstract Cancer-associated fibroblasts (CAFs) have emerged as a dominant non-hematopoietic cell population in the tumour microenvironment, serving diverse functions in tumour progression. However, the mechanisms via which CAFs influence the anti-tumour immunity remain poorly understood. Here, using multiple tumour models and biopsies from cancer patients, we report that α-SMA+ CAFs can form immunological synapses with Foxp3+ regulatory T cells (Tregs) in tumours. Notably, α-SMA+ CAFs can phagocytose and process tumour antigens and exhibit a tolerogenic phenotype which instructs movement arrest, activation and proliferation in Tregs in an antigen-specific manner. Moreover, α-SMA+ CAFs display double-membrane structures resembling autophagosomes in their cytoplasm. Single-cell transcriptomic data showed an enrichment in autophagy and antigen processing/presentation pathways in α-SMA-expressing CAF clusters. Conditional knockout of Atg5 in α-SMA+ CAFs promoted inflammatory re-programming in CAFs, reduced Treg cell infiltration and attenuated tumour development. Overall, our findings reveal an immunosuppressive mechanism entailing the formation of synapses between α-SMA+ CAFs and Tregs in an autophagy-dependent manner."

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ePhective (San Francisco), a newly founded phage therapy company started by Joe Bondy-Denomy & Zemer Gitai, is looking for 2 Phage Scientists. Phage Scientist ePhective See the full job description on jobRxiv: https://lnkd.in/dZ_jWTfH #bacterial_and_viral_infection #bacterial_pathogenesis #bacterial_host_interaction #bacteriology #bacteriophages

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ePhective (San Francisco), a newly founded phage therapy company started by Joe Bondy-Denomy & Zemer Gitai, is looking for 2 Phage Scientists. Phage Scientist ePhective See the full job description on jobRxiv: https://lnkd.in/dtyePUrh #bacterial_and_viral_infection #bacterial_pathogenesis #bacterial_host_interaction #bacteriology #bacteriophages

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I would be happy to contribute to the course/workshop where I can share my own expertise and experience working with CRISPR-Cas and toxic nucleases in exchange to learning more about Cryo-EM and Cryo-ET (all from sample prep to data processing) - if interested, let me know! 😊 *comic from the book "Jeff's View: on Science and Scientists"

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Weekly digest breakthroughs in cancer treatment, chemistry, and more by The Babak Lab https://lnkd.in/deJEF24c Maria (Masha) Babak, PhD FDA Berkeley Lab Vanessa Barrs #Cancer #CancerTreatment #FDA #CancerEducation #ScientificBreakthroughs #OncoDaily #Oncology

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Our very own Prof. Hermona Soreq, Endowed Slesinger Professor of Molecular Neuroscience, is featured in the "Brain Medicine" journal. Prof. Soreq's groundbreaking work on poly(A) tails, mRNA stability, and acetylcholine is revolutionizing our understanding of brain-body communication across the lifespan. Her research sheds light on stress responses, neurodegenerative diseases, and the intricate workings of the cholinergic system (= one of the most important modulatory neurotransmitter systems in the brain). We invite you to read the below article, which showcases Prof. Soreq's pioneering contributions to molecular neuroscience and her ongoing impact on the field. Edmond and Lily Safra Center for Brain Sciences - ELSC #HebrewUniversity #Neuroscience #BrainResearch #MolecularBiology

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In @NatureBiotech: @ArunJSanyalVCU and @VCUMassey colleagues reveal a new genetic code that recruits and deploys tumor cells to incite a biological turf war by invading healthy organs and overpowering the normal cells. #livertwitter https://bit.ly/4gtxy43

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