Dr. Rory (they/them)

Molecular Therapy Presents: Innovations in Aptamer Technology In 1990, two seminal studies introduced a novel method, systematic evolution of ligands by exponential enrichment (SELEX), to discover and isolate…

Don't miss the next #MolecularTherapy Presents webinar, Innovations in Aptamer Technology (December 4 at 1 PM CST). Come learn about the most cutting-edge applications of aptamers. Register here today:

Generating CAR T cells in vivo with lentiviral vectors with Drs. Rachael Nimmo and Kyriacos Mitrophanous - ASGCT Podcast Network Editor-in-Chief of Molecular Therapy, Dr. Joseph Glorioso, is joined by Rachael Nimmo, PhD, former director of the Cell Technology Group at Oxford Biomedica, and Kyriacos Mitrophanous, PhD, Chief Innovation Officer at Oxford Biomedica, to discuss an article recently published in Molecular Therapy by Nimmo and colleagues titled “Efficient in vivo generation of CAR T cells using a retargeted fourth-generation lentiviral vector.” Music: 'Electric Dreams' by Scott Buckley - released under CC-BY 4.0. www.scottbuckley.com.au

New #MolecularTherapy podcast: Generating CAR T cells in vivo with lentiviral vectors with Drs. Rachael Nimmo and Kyriacos Mitrophanous
Listen now:

On the cover: Chan, Wong, and colleagues depict how epigen improves diabetes via pancreas, liver, and adipose tissue. Image credit: Ka-Ying Chan and Zuoze Hao.

The November issue of #MolecularTherapy is out now! Dive into the latest in #immunotherapy, #GeneTherapy, commercially pre-viable CGTs for rare diseases, and #GeneEditing. Read it now: http://dlvr.it/TP7vtZ
#ASGCT

Molecular Therapy Presents: Advancing AAV Technology The use of AAV, the gold-standard viral vector for gene therapy, has successfully progressed from preclinical studies to multiple FDA-approved therapies.…

TODAY: #MolecularTherapy Presents has an exciting webinar, Advancing #AAV Technology, at 10-11 AM CDT. Don't miss out on this free event; register now: http://dlvr.it/TNqHBN
#ASGCT #genetherapy 🧬

Molecular Therapy Presents: Advancing AAV Technology The use of AAV, the gold-standard viral vector for gene therapy, has successfully progressed from preclinical studies to multiple FDA-approved therapies.…

#MolecularTherapy Presents has an exciting webinar, Advancing #AAV Technology, on October 22 at 10-11 AM CDT with Drs. Çağla Aksu Küz and Susumu Uchiyama. Don't miss out; register today: http://dlvr.it/TNhYj4
#ASGCT #genetherapy 🧬

Molecular Therapy Presents: Advancing AAV Technology The use of AAV, the gold-standard viral vector for gene therapy, has successfully progressed from preclinical studies to multiple FDA-approved therapies.…

#MolecularTherapy Presents has an exciting webinar, Advancing #AAV Technology, on October 22 at 10-11 AM CDT with Drs. Çağla Aksu Küz and Susumu Uchiyama. Don't miss out; register today: http://dlvr.it/TNSBsh
#ASGCT #genetherapy 🧬

The October 2025 issue of #MolecularTherapy features an #AAV #gene therapy for LCA5-associated Leber congenital amaurosis, replicon #RNAvaccines for #HIV, and in vivo generation of #CAR_T cells. Read it here: http://dlvr.it/TNQ1L7

Chao and colleagues show an image of glioblastoma (red) treated with CF17 oncolytic virus (green). Image credit: Jianfei Chao.

#MolecularTherapy Oncology's Sept 2025 issue covers new research on #Nanobodies, #OncolyticViruses, #BaseEditing, and #TherapeuticTargets in #Cancer. Check it out today: http://dlvr.it/TN8jsh
#ASGCT

Molecular Therapy Presents: Advancing AAV Technology October 22, 2025 | 10:00 AM CT

Join our free #MolecularTherapy Presents webinar to explore the latest in #AAV manufacturing and capsid design for #genetherapy. Register here today: http://dlvr.it/TN6NQT
#ASGCT

Direct optogenetic activation of skeletal muscle offers an alternative to invasive electrical stimulation. Knapman et al. tested the ability of adeno-associated viral vectors (AAV9 and AAVMYO) to confer light sensitivity to upper airway muscles in a model of obstructive sleep apnea. The image shows robust opsin expression (orange) in axial sections from the anterior and middle regions of a rat tongue 3 weeks after intramuscular AAV9 delivery. Image credit: Fiona L. Knapman.

Check out the latest issue of #MolecularTherapy Methods & Clinical Development for reviews on #LNPs and #genetherapy redosing, plus research on #AAV manufacturing, #siRNA therapy, and #celltherapy. Read it here: http://dlvr.it/TN2q33
#MTMCD

Molecular Therapy podcast: Lipid nanoparticles with Drs. Daniel Stone, Harrison Dulin, and Keith R. Jerome

Check out the latest episode of the #MolecularTherapy podcast on #lipidnanoparticles featuring Drs. Stone, Dulin, and Jerome from Fred Hutchinson Cancer Center. Listen now: http://dlvr.it/TN0b93

The cover image shows pumpkin spice DNA. Formulation is a key component in the success of nucleic acid delivery. Sallah et al. investigated how the addition of cucurbit[n]urils (CBs) to cationic polymers altered the profile of DNA delivery, reducing inflammation but increasing expression. Image credit: John Tregoning, Charlotte Tregoning, and Sophie Tregoning.

The new issue of #MolecularTherapy Nucleic Acids is out now. This issue features original articles on replicon #RNA vaccination, #circRNA therapeutics, mRNA-encoded BiTE cancer therapy, and more. Read it today: http://dlvr.it/TMzM2n
#MTNA #ASGCT

New issue of Molecular Therapy out now!

Out now: the September 2025 issue of Molecular Therapy features new research on in vivo base editing, stem cell therapy, microglial replacement, and more. Read the new issue today: www.cell.com/molecular-th...

Development of a Novel Ionizable Lipid-Based mRNA Vaccine for Broad Protection Against Streptococcus pneumoniae Shi Xu and colleagues report a novel ionizable lipid-based LNP system that exhibits high mRNA delivery efficiency and develop a phylactic mRNA vaccine providing cross-protection against multiple serotypes of Streptococcus pneumoniae. This work advances the development of mRNA vaccine for combating bacterial infections.

#MTNA

25 years of Molecular Therapy with Drs. Rayne Rouce and Joseph Glorioso

The newest episode of the #MolecularTherapy podcast is out now and covers the topic of democratizing gene and cell therapy with guest Dr. Rayne Rouce. Listen here: http://dlvr.it/TMS5LN

Molecular Therapy Presents: Advancements in Pediatric Cancer Therapy | ASGCT - American Society of Gene & Cell Therapy

TODAY: #MolecularTherapy Presents: Advancements in Pediatric Cancer Therapy at 1 PM CT. You will learn more about therapies for pediatric AML, bone sarcomas and rare pediatric cancers.
Register now:

Molecular Therapy Presents: Advancements in Pediatric Cancer Therapy | ASGCT - American Society of Gene & Cell Therapy

Have you registered for next week's #MolecularTherapy presents webinar on #PediatricCancer therapy yet? Check out our great lineup of speakers and register here:

Molecular Therapy Oncology | Cell Press From the first use of chemotherapy in the 1960s to the first FDA-approved CAR T-cell therapy in 2017, the treatment of pediatric cancers has evolved to become safer and more effective, increasing survival rates from 10% to nearly 80%. Despite these pivotal successes, cancer remains a leading cause of death among children and adolescents. This special issue is focused on the history, current state, and future of pediatric cancer therapy. We invite authors to submit research including the following treatment modalities of interest: Gene therapies Cell-based therapies Molecular therapies Viral therapies Precision oncology Vaccines Antibodies Immune checkpoint inhibitors Submission due date: September 30, 2025 This special issue will include invited reviews on the following topics: Current and emerging strategies for prevention of relapse post-stem cell transplant in children and young adults with acute leukemia Hema Rangarajan, MD, and Alex Rankin, MD, Nationwide Children's Hospital Cancer-associated fibroblasts (CAFs) in pediatric solid tumors Julie Tomolonis, MD, PhD, Elizabeth Garfinkle, PhD, Elaine Mardis, PhD, and Timothy Cripe, MD, PhD, Nationwide Children's Hospital Current landscape of CAR T-cell therapy in children and young adults with B-cell acute lymphoblastic leukemia Sara Silbert, MD, MHSc, Alexandra Dreyzin, MD, Flavia Mesquita Gava, MD, Chelsea O’Koren, MD, and Julie Ma, MD, National Cancer Institute Limitations of preclinical modeling in CAR T cells Victoria Giordani, MD, Julie Ma, MD, Josquin Moraly, MS, MD, Rosa Nguyen, MD, PhD, and Brynn Duncan, MD, National Cancer Institute Updates on cell therapy for AML Anand Bhagwat, MD, PhD, Children's Hospital of Philadelphia and Swati Naik, MBBS, St. Jude Children's Research Hospital Advances in immune-based therapies for pediatric bone sarcomas Brian Ladle, MD, PhD, and Nico Llosa, MD, Johns Hopkins University School of Medicine Advances in pediatric oncology: Challenges and opportunities in the low- and middle-income countries Chetan Dhamne, MD, Ramandeep Arora, MBBS, DCH, MRCPCH, CCT, MD, FRCPCH, Rahul Purwar, PhD, and Gaurav Narula, MD, Tata Memorial Hospital Adoptive cell therapies for CNS tumors: Current landscape and future directions Kun-Wei Song, MD, Aanchal Preet Kaur, PhD, and Sneha Ramakrishna, MD, Stanford School of Medicine Precision medicine in pediatric oncology: Current applications and future directions Nilay Shah, MD, Nationwide Children's Hospital and The Ohio State University College of Medicine Updates on rare pediatric cancer Jacquelyn Crane, MD, Children's Hospital of Philadelphia, Robyn Gartrell, MD, Johns Hopkins University School of Medicine, and Kris Ann Schultz, MD, Children's Minnesota, and Theodore W. Laetsch, MD, Children's Hospital of Philadelphia

#MolecularTherapy Oncology has an active call for papers for a special issue on Advancements in Pediatric Cancer Therapy. Learn more and submit your paper before September 30, 2025:

Molecular Therapy Presents: Advancements in Pediatric Cancer Therapy. Wednesday, August 6, 2025 at 1 PM CT.

Join us for this free webinar on August 6th hosted by #ASGCT and learn more about #PediatricCancerTherapy:
http://dlvr.it/TM2Ln3

25 Years of Molecular Therapy with Drs. Beverly Davidson and Jeff Chamberlain - ASGCT Podcast Network This episode is the third in a series hosted by Molecular Therapy Editor-in-Chief Joseph Glorioso, PhD, that will cover comprehensive reviews of critical developments in the field of gene and cell therapy over the past 25 years.   In this episode, Dr. Glorioso will discuss the reviews, “Current trends in gene therapy to treat inherited disorders of the brain,” with author Beverly Davidson, PhD, The Children’s Hospital of Philadelphia, and “The road toward AAV-mediated gene therapy of Duchenne muscular dystrophy,” with author Jeff Chamberlain, PhD, University of Washington School of Medicine.  If you enjoy this deep dive into research that is critical to the field, check out two more ASGCT events happening this fall: Advancing Cell and Gene Therapies for Cancer and Breakthroughs in Targeted In Vivo Gene Editing. During these events – in October and November, respectively – you’ll be able to hear directly from researchers about their own work related to these topics, or you can submit your own abstract for possible presentation. We hope to see you there! Music: 'Electric Dreams' by Scott Buckley - released under CC-BY 4.0. www.scottbuckley.com.au 

Out now: Drs. Beverly Davidson and Jeff Chamberlain discuss gene therapy for inherited brain diseases and Duchenne muscular dystrophy on the #MolecularTherapyPodcast:

Child's drawing of cancer therapies and cancer cells surrounding a heart containing a gold ribbon, the international symbol for childhood cancer awareness.

Register today for Molecular Therapy Presents: Advancements in pediatric cancer therapy
Topics: Cell therapy for AML and Updates on rare pediatric cancer
Date/time: August 6, 2025 at 1 PM CT
http://dlvr.it/TLrm7S

Yang et al. demonstrated that following acute kidney injury (AKI), CD38hi macrophages, derived from resident macrophages via Csf1 signaling, secrete the NAD-depleting enzyme CD38, inducing senescence in renal tubular cells and promoting chronic inflammation and renal fibrosis. The image depicts CD38hi macrophages (symbolized by wood-consuming termites) inducing tubular cell senescence (represented as tree withering) through metabolic depletion of NAD+ pools (metaphorized as essential tree nutrients). Image credit: Weijian Yao.

The July 2025 issue of #MolecularTherapy is out now. This issue features articles about LNP targeting, lentiviral gene therapy, and more. Read it today: http://dlvr.it/TLrVcg

Interested in learning more about #AAV design and manufacturing? Check out #MolecularTherapy Methods & Clinical Development's new special collection, Advancing AAV Technology - Part One:
http://dlvr.it/TLJ8g7

The American Society of Gene & Cell Therapy logo

A new episode of the ‪@moltherapy.bsky.social‬ podcast just dropped. Listen to 25 years of Molecular Therapy with Sheila Mikhail and Dr. Mark Kay:
http://dlvr.it/TLHsX4

YouTube video by U.S. Food and Drug Administration FDA Roundtable on Cell and Gene Therapy

Thank you to the @fda.gov, Dr. Vinay Prasad, + Dr. Marty Makary for hosting today’s #CGT roundtable. ASGCT’s Dr. Terry Flotte + Dr. Paula Cannon joined experts in a rich discussion on advancing CGT. Tune in at 13:07 + 1:07:25: www.youtube.com/watch?v=0qDh...

#GeneTherapy #CellTherapy

The cover image depicts bispecific antibodies tethering mRNA-containing lipid nanoparticles (LNPs) to surface proteins on cells.

The June 2025 issue of Molecular Therapy Nucleic Acids is out now. Read it today:
http://dlvr.it/TLH7C8

A DNA double helix in a capsule surrounded by cells. Credit: Vink Fan/Shutterstock.

#MolecularTherapy's Clinical gene and cell therapy special issue is a must-read comprehensive update on AAV, CAR T-cell, NK cell, RNA, and genome editing therapies (and much more) in the clinic. Read more here: http://dlvr.it/TLBbkl

Molecular Therapy Presents: 25th Anniversary Special Issue. Wednesday, April 23, 2025 at 11 AM CT

In honor of Molecular Therapy's 25th anniversary, EiC Dr. Joseph Glorioso is hosting a free webinar with 3 leading authors who have contributed to MT's success. Learn more and register here today: bit.ly/3E7uhKm

#MolecularTherapy #FreeWebinar #GeneTherapy #CellTherapy #ASGCT

Trump hits NIH with ‘devastating’ freezes on meetings, travel, communications, and hiring Researchers facing “a lot of uncertainty, fear, and panic”

I've been trying to remain optimistic and not panic about the barrage of orders and hiring freezes from the new administration, but this seems beyond the usual "new leadership, new policies":

www.science.org/content/arti...