Case Studies in Translational Research

Bird Flu Outbreak in Dairy Cows Is Widespread, Raising Public Health Concerns

Highly Pathogenic Avian Influenza A(H5N1) Virus Infection in a Dairy Farm Worker

G. Brandon Atkins, MD, PhD

Targeting factor XI and factor XIa to prevent thrombosis

Factor XI Inhibition to Uncouple Thrombosis From Hemostasis

Anticoagulation in patients with kidney failure on dialysis: factor XI as a therapeutic target

Optional: Emerging Therapies in Thrombosis: Factor XI/XIa Inhibitors

Drew Weissman, MD, PhD

In vivo hematopoietic stem cell modification by mRNA delivery

Base editing of hematopoietic stem cells rescues sickle cell disease in mice

Risk of Second Tumors and T-Cell Lymphoma after CAR T-Cell Therapy

Indolent CD4+ CAR T-Cell Lymphoma after Cilta-cel CAR T-Cell Therapy

Enabling non-viral DNA delivery using lipid nanoparticles co-loaded with endogenous anti-inflammatory lipids

Lipid Nanoparticle-Associated Inflammation is Triggered by Sensing of Endosomal Damage: Engineering Endosomal Escape Without Side Effects

Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant (Supplementary Appendix)

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

David Fajgenbaum, MD, MSc

Identifying and targeting pathogenic PI3K/AKT/mTOR signaling in IL-6 blockade–refractory idiopathic multicentric Castleman disease

KGML-xDTD: a knowledge graph–based machine learning framework for drug treatment prediction and mechanism description

Implementing a Real-Time Electronic Data Capture System to Improve Clinical Documentation in Radiation Oncology

A Machine Learning Model Approach to Risk-Stratify Patients With Gastrointestinal Cancer for Hospitalization and Mortality Outcomes

Artificial intelligence models to analyze cancer images take shortcuts that introduce bias

FDA Software as a Medical Device Webpage

Christina Coughlin, MD, PhD

Driving Diversity and Inclusion in Cancer Drug Development – Industry and Regulatory Perspectives, Current Practices, Opportunities, and Challenges

Investigational New Drug (IND) Application | FDA

Phase 3 Trial of Transplantation of Human Islets in Type 1 Diabetes Complicated by Severe Hypoglycemia

Developments in stem cell-derived islet replacement therapy for treating type 1 diabetes