Scientist, professor, entrepreneur
Joined May 2020
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Chris Doering retweeted
A new study published in the New England Journal of Medicine tested a groundbreaking #GeneTherapy using a lentiviral vector for people with severe #hemophilia A. The evidence for the safety and efficacy are promising. Read more: bit.ly/4izgITA #BleedingDisorders
Chris Doering retweeted
Congrats to Trent Spencer, PhD, who led the first-in-human clinical study on a new gene therapy for severe hemophilia A with Alok Srivastava, MD. Published in @NEJM, results showed stable factor VIII expression in patients. Learn more: nejm.org/doi/full/10.1056/NE….
Chris Doering retweeted
Five patients with hemophilia A had dramatic reductions in bleeding episodes after undergoing autologous HSCT with lentiviral vector–transduced cells encoding a CD68-targeted factor VIII transgene. Full trial results: nej.md/3Vk5xUA
#ASH24
ALT Panel A: A graph of the factor VIII activity in each of five participants after they received gene therapy with CD68-ET3-LV–transduced autologous CD34+ hematopoietic stem cells. Panel B: A graph of the number of bleeding events for each participant before and after gene therapy.
Chris Doering retweeted
Groundbreaking first-in-human study published in NEJM, and presented at ASH 2024 - Lentiviral gene therapy in Hemophilia A. Congrats Dr. Alok Srivastava and team @cmcvelloreoff @OffCMCVellore
Chris Doering retweeted
Congrats to #ASH2024 Hematology Inclusion Pathway Graduate Student Abstract Achievement Award Recipients: Alejandro De Janon (Advisors: Dr. @douglaskgraham and Dr. Takayama) and Kristopher A. Knight (Advisors: Dr. Raikar and Dr. Doering)! They will present their posters at 6 PM.
Chris Doering retweeted
Stable FVIII levels after lentiviral gene therapy in hemophilia A. Results of small phase 1 study from Vellore in India will be presented at #ASH2024. Yes it is small numbers with short follow up, but it shows that stable FVIII levels of >15% can be achieved. This is the first gene therapy that can be used in children with #hemophilia. However, it requires an autologous bone marrow transplant, the lentiviral delivered genes integrate and we are at a time of safe alternative therapies.
Chris Doering retweeted
Period of severe cytopenias were short. FVIII levels kept between 50-100% with EHL-FVIII until engraftment.
Chris Doering retweeted
High FVIII levels after ex vivo #genetherapy for #hemophilia A. Does require bone marrow conditioning, but no immunosuppressive after discharge.
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Chris Doering retweeted
By probing evolutionary landscape of FIX,
we mapped and functionally characterized combination of ancestral amino acid substitutions
▶️capable of enhancing potency of human FIX and FIX-Padua, 50x and 10x, respectively.
@ChrisDoering4 @SpiegelClint
jthjournal.org/article/S1538…
Chris Doering retweeted
First in human clinical work in Hemophilia A gene therapy using alternative strategy- ex vivo hematopoietic cells (with the expression in differential gene) using the CD68 ET3 LV HSC for FVIII expression#ASH23 reported by prof Srivastava
Chris Doering retweeted
Continuing this morning’s great session at #ISTH2021 on Structural Determinants of Function, Clint Spiegel (@SpiegelClint) just gave a beautifully clear and engaging talk on “Factor VIII Structure: Determinants of Inhibitor Development.”
Chris Doering retweeted
Check out our preprint characterizing the solution structure of the intrinsic tenase complex!
SAXS analysis of the intrinsic tenase complex bound to lipid nanodisc highlights intermolecular contacts between factors VIIIa/IXa biorxiv.org/cgi/content/shor… #bioRxiv
Chris Doering retweeted
Great science coming out out of @ATLPedsHemOnc @AtlPedsResearch
New #coverart #July selected by EIC @JHMorrissey @DavidLillicrap Zimowski et al @EmoryUniversity find novel FV variant, deletion in exon 13➡️ alternative splicing. FV-short & TFPIalpha plasma levels similar to East Texas #bleeding disorder + commentary. bit.ly/3AqhtYY
Chris Doering retweeted
New article: Elucidating the molecular pathogenesis underlying East Texas #bleeding disorder led to discovery of alternatively spliced F5 transcripts. Zimowski et al @ATLPedsHemOnc identify novel heterozygous 832 bp deletion. Commentary by Castoldi.
bit.ly/2ShPqJV
