Research / Cell and Gene Therapy

Cell and Gene Therapy

Delivering genes and gene editors into hard-to-transfect cells and tissues.

Getting genes and gene editors into the right cells, safely and at scale, is one of the main bottlenecks for advanced therapies. Nanoneedles deliver nucleic acids and proteins into cells with minimal disruption, both in culture and in tissue.

  • Gene editing for inherited disease. Nanoneedles delivered an adenine base editor that corrected a pathogenic COL7A1 variant in cells from people with recessive dystrophic epidermolysis bullosa with 96.5% efficiency (Advanced Materials, 2025). With Cure EB, the NanoCure project is preparing a nanoneedle bandage for a first-in-human study. We also work on gene editing for inherited eye disease.
  • Cell therapy manufacturing. Nanoneedle electroporation engineers primary human regulatory T cells with chimeric antigen receptors without viral vectors (Advanced Science, 2025), and our perfusion microbioreactor supports small-batch CAR-Treg production (iScience, 2026).
  • How it works. We found that caveolar endocytosis is what turns nanoneedle delivery into gene expression (ACS Nano, 2026).
False-coloured electron micrograph of a pink cell sitting on green nanoneedles.

Recent publications

Immune reprogramming of 3D tumor models via optoporation-mediated targeted gene delivery to macrophages

Poljak I, Hussein IN, Gu C, Giustarini G, Teng X, Toyama Y, Chiappini C, Adriani G

PreprintbioRxiv (Cold Spring Harbor Laboratory) · 2026

Perfusion microbioreactor for CAR-Treg manufacturing

Edwards W, Sun N, Wang Y, Lu Y, Wang C, Mastronicola D, Scottà C, Romano M, Cejas CM, Espinet A, Lombardi G, Chiappini C

iScience · 2026 · vol. 29, no. 4, pp. 115246

Caveolar Endocytosis Governs Nanoneedle Transfection

Sun N, Wang C, Wang Y, Edwards W, Dimitrievska M, Li Y, Vasovic N, McLennan S, Zhu H, Mäkilä E, Salonen J, …, Chiappini C

ACS Nano · 2026 · vol. 20, no. 6, pp. 4663–4676 · Open access version

Nanoneedle-Based Electroporation for Efficient Manufacturing of Human Primary Chimeric Antigen Receptor Regulatory T-Cells

Sun N, Wang C, Edwards W, Wang Y, Lu X, Gu C, McLennan S, Shangaris P, Qi P, Mastronicola D, Scottà C, …, Chiappini C

Advanced Science · 2025 · vol. 12, no. 21, pp. 2416066 · Open access version

Porous Silicon Nanoneedles Efficiently Deliver Adenine Base Editor to Correct a Recurrent Pathogenic COL7A1 Variant in Recessive Dystrophic Epidermolysis Bullosa

Mustfa SA, Dimitrievska M, Wang C, Gu C, Sun N, Romańczuk K, Karpinski P, Łaczmański Ł, McGrath JA, Jacków-Malinowska J, Chiappini C

Advanced Materials · 2025 · vol. 37, pp. e2414728

Spatially-Resolved Organoid Transfection by Porous Silicon-Mediated Optoporation

Spiteri C, Caprettini V, Wang Y, Dominguez-Gil S, Kaasalainen M, Wang C, Martella DA, McLennan S, Vashisth P, Gary-Bobo M, Nguyen C, …, Chiappini C

Advanced Materials · 2024 · vol. 36, no. 49, pp. 2407650 · Open access version

Integrating Porous Silicon Nanoneedles within Medical Devices for Nucleic Acid Nanoinjection

Wang C, Gu C, Popp C, Vashisth P, Mustfa SA, Martella DA, Spiteri C, McLennan S, Sun N, Riddle M, Eide CR, …, Chiappini C

ACS Nano · 2024 · vol. 18, no. 23, pp. 14938–14953

CRISPR/Cas-Assisted Nanoneedle Sensor for Adenosine Triphosphate Detection in Living Cells

Kim H, Gu C, Mustfa SA, Martella DA, Wang C, Wang Y, Chiappini C

ACS Applied Materials and Interfaces · 2023 · vol. 15, no. 43, pp. 49964–49973 · Open access version

All Cell and Gene Therapy publications