In Vivo Gene Therapy Core

The In Vivo Gene Therapy Core is dedicated to advancing innovative in vivo gene and cell therapy approaches for genetic diseases affecting the lung, heart, blood, and other organ systems. Using targeted delivery technologies, including tissue-targeted nanoparticles, viral vectors, and emerging platforms, the Core aims to develop safe, scalable therapeutic strategies that can accelerate clinical translation and expand access to advanced therapies worldwide, including in low-resource settings.

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photo of Naoya Uchida

Research

Rapid advances in lipid nanoparticle, viral vectors, and gene delivery technologies are creating new opportunities to deliver genetic therapies directly in vivo. The Core supports investigators in addressing key translational challenges, including: (1) identifying and optimizing tissue-targeted delivery molecules, (2) engineering precise gene delivery systems, (3) evaluating therapeutic efficacy and safety in animal models, and (4) advancing promising approaches toward clinical applications.

Through these efforts, the Core aims to accelerate translational research and generate the preclinical evidence needed to advance safe, scalable therapies from early development toward IND-enabling studies and clinical application, with the ultimate goal of improving patient care.

Major Services and Equipment

Services

  1. Consultation and Project Assessment
    • Evaluation of in vivo gene therapy concepts and preliminary data
    • Guidance on translational strategy and experimental design
  2. Vector and Nanoparticle Development
    • Lentiviral vector design
    • Lipid nanoparticle design and formulation
    • Development of gene editing strategy
    • Screening and development of tissue-targeted delivery platforms
  3. Preclinical Evaluation
    • Evaluation of gene delivery, biodistribution, and therapeutic efficacy in animal models
  4. Planned Future Translational Support
    • Preclinical safety assessments in animal models
    • Support for investigational new IND-enabling studies and regulatory strategy development

Instruments

  • Azure 600 Imaging System
  • Bio-Rad QX600 Droplet Digital PCR System
  • Cytiva NanoAssemble Ignite Nanoparticle Formulation System
  • Cytiva NanoAssemblr Spark Nanoparticle Formulation System
  • Cytiva VIA Freeze Duo Controlled-Rate Freezers
  • Horiba ViewSizer 3000
  • Labconco FreeZone 2.5 Liter -84C Benchtop Freeze Dryer
  • Malvern Panalytical Zetasizer Advance Ultra Red
  • MaxCyte Atx Electroporator
  • Molecular Devices SpectraMax i3x Multi-Mode Microplate Reader
  • Thermo QuantStudio 6 Pro Real-Time PCR
  • Thermo LYNX 6000 Superspeed Centrifuge

Featured Publications

Fertility-preserving myeloablative conditioning using single-dose CD117 antibody-drug conjugate in a rhesus gene therapy model

Authors: Uchida N, Stasula U, Demirci S, Germino-Watnick P, Hinds M, Le A, Chu R, Berg A, Liu X, Su L, Wu X, Krouse AE, Linde NS, Bonifacino A, Hong SG, Dunbar CE, Lanieri L, Bhat A, Palchaudhuri R, Bennet B, Hoban M, Bertelsen K, Olson LM, Donahue RE, Tisdale JF
Nat Commun 2023 Oct 12;14(1):6291
Read Publication on Fertility-preserving myeloablative conditioning using single-dose CD117 antibody-drug conjugate in a rhesus gene therapy model

Preclinical evaluation for engraftment of CD34+ cells gene-edited at the sickle cell disease locus in xenograft mouse and non-human primate models

Authors: Uchida N, Li L, Nassehi T, Drysdale CM, Yapundich M, Gamer J, Haro-Mora JJ, Demirci S, Leonard A, Bonifacino AC, Krouse AE, Linde NS, Allen C, Peshwa MV, De Ravin SS, Donahue RE, Malech HL, Tisdale JF
Cell Rep Med 2021 Apr 20;2(4):100247
Read Publication on Preclinical evaluation for engraftment of CD34+ cells gene-edited at the sickle cell disease locus in xenograft mouse and non-human primate models

Sustained fetal hemoglobin induction in vivo is achieved by BCL11A interference and coexpressed truncated erythropoietin receptor

Authors: Uchida N, Ferrara F, Drysdale CM, Yapundich M, Gamer J, Nassehi T, DiNicola J, Shibata Y, Wielgosz M, Kim YS, Bauler M, Throm RE, Haro-Mora JJ, Demirci S, Bonifacino AC, Krouse AE, Linde NS, Donahue RE, Ryu B, Tisdale JF
Sci Transl Med 2021 Apr 28;13(591):eabb0411
Read Publication on Sustained fetal hemoglobin induction in vivo is achieved by BCL11A interference and coexpressed truncated erythropoietin receptor

Meet the Team

photo of Naoya Uchida

Naoya Uchida, M.D., Ph.D.

Core Director

Naoya Uchida is the Director of the In Vivo Gene Therapy Core at the National Heart, Lung, and Blood Institute. His research focuses on developing ex vivo and in vivo hematopoietic stem cell-targeted therapies using lentiviral vectors, genome editing, and targeted delivery technologies. His work spans therapeutic design, preclinical animal studies, and clinical translation.