journal article·2026-07

Enhancing in vivo B cell gene delivery using targeted lentiviral vectors and transduction enhancers

Marshall Fritz, Limei Shen, Lucas C. Olson, Jingyu Zhao, Madison Card, Raymond Pickles, Samuel K. Lai

Acta Biomaterialia· Volume 218
Publisher page

Abstract

B cells occupy a distinct niche in humoral immunity, and engineered B cells that leverage their unique biological functions hold significant promise for therapeutic applications. Here, we combine chemical and virological strategies to establish a method for in situ engineering of B cells by optimizing formulations of CD20-targeted Nipah paramyxovirus-pseudotyped lentiviral vectors (LVs) coupled with the use of transduction enhancers (TEs). Select TE combinations enabled greater than 3-fold improvements in transduction efficiency of isolated B cells as well as B cells within human PBMC populations in vitro, without compromising targeting specificity. In particular, a combination of poloxamer 407 and valproic acid, as well as a formulation containing four distinct TEs, significantly enhanced transduction of circulating human B cells in PBMC-engrafted NSG mice compared with no-TE controls. This enhancement resulted in more than a 20-fold increase in serum levels of a recombinant anti-RSV antibody by day 26, with no detectable safety concerns or off-target transduction. Together, these findings demonstrate that appropriate formulation of LVs with selected TEs enables effective and specific in vivo B-cell engineering, supporting the development of new B-cell-based therapeutic strategies that integrate gene and cell therapy approaches to reprogram immunity. STATEMENT OF SIGNIFICANCE: Efficient in vivo cell engineering remains limited by two major challenges: achieving cell-type specificity and attaining sufficient gene transfer efficiency for therapeutic relevance. This study demonstrates that formulation of targeted lentiviral vectors with well-characterized transduction enhancers can substantially improve in vivo gene delivery to B cells without compromising specificity. By combining CD20-targeted paramyxovirus-pseudotyped lentiviral vectors with selected small-molecule and polymer-based enhancers, we achieve markedly enhanced transduction of human B cells in vitro and in vivo. Given the intrinsic difficulty of transducing circulating B cells, these findings establish a broadly applicable strategy for improving targeted gene delivery through vector formulation.
Source: europepmc · Quality: candidate unverified · Source record ↗

Research context

OpenAlex topics: Virus-based gene therapy research · RNA Interference and Gene Delivery · CRISPR and Genetic Engineering

SciVX discovery tags are terminology-based. OpenAlex topics are source-provided classifications.

References (49)

Show source-provided references
1. B cell responses: cell interaction dynamics and decisions10.1016/j.cell.2019.03.016 ↗
2. Neutralizing antibodies derived from the B cells of 1918 influenza pandemic survivors10.1038/nature07231 ↗
3. Baboon envelope pseudotyped lentiviral vectors efficiently transduce human B cells and allow active factor IX B cell secretion in vivo in NOD/SCIDγc-/- mice10.1111/jth.13520 ↗
4. Generation of stable monoclonal antibody–producing B cell receptor–positive human memory B cells by genetic programming10.1038/nm.2071 ↗
5. Molecular design, optimization, and genomic integration of chimeric B cell receptors in murine B cells10.3389/fimmu.2019.02630 ↗
6. Immune modulatory cell therapy for hemophilia B based on CD20-targeted lentiviral gene transfer to primary B cells10.1016/j.omtm.2017.03.005 ↗
7. Challenges and opportunities in gene editing of B cells10.1016/j.bcp.2022.115285 ↗
8. Price and prejudice? The value of chimeric antigen receptor (CAR) T-cell therapy10.3390/ijerph191912366 ↗
9. Efficient culture of Human naive and memory B cells for use as APCs10.4049/jimmunol.1502193 ↗
10. Surface-engineered lentiviral vectors for selective gene transfer into subtypes of lymphocytes10.1016/j.omtm.2018.10.006 ↗
11. Mystery solved: VSV-G-LVs do not allow efficient gene transfer into unstimulated T cells, B cells, and HSCs because they lack the LDL receptor10.1182/blood-2013-11-540641 ↗
12. The ins and outs of antigen uptake in B cells10.3389/fimmu.2022.892169 ↗
13. Carneiro · 2016
14. Receptor-targeted Nipah virus glycoproteins improve cell-type selective gene delivery and reveal a preference for membrane-proximal cell attachment10.1371/journal.ppat.1005641 ↗
15. Targeted cell entry of lentiviral vectors10.1038/mt.2008.128 ↗
16. Lentiviral vectors with measles virus glycoproteins – dream team for gene transfer?10.1016/j.tibtech.2009.02.002 ↗
17. Protransduzin™ - A novel transduction enhancer to accelerate en gros CAR-T production10.1016/j.jcyt.2019.04.044 ↗
18. Effects of polybrene and retronectin as transduction enhancers on the development and phenotypic characteristics of VHH-based CD19-redirected CAR T cells: a comparative investigation10.1007/s10238-022-00928-8 ↗
19. Improving CAR T cell therapy by optimizing critical quality attributes10.1053/j.seminhematol.2020.07.005 ↗
20. Optimized lentiviral transduction protocols by use of a poloxamer enhancer, spinoculation, and scFv-antibody fusions to VSV-G10.1007/978-1-4939-3753-0_4 ↗
21. High-efficiency lentiviral transduction of Human CD34+ cells in High-density culture with poloxamer and prostaglandin E210.1016/j.omtm.2019.01.005 ↗
22. Vectofusin-1 improves transduction of primary Human cells with diverse retroviral and lentiviral pseudotypes, enabling robust, automated closed-system manufacturing10.1089/hum.2019.157 ↗
23. Vectofusin-1, a potent peptidic enhancer of viral gene transfer forms pH-dependent α-helical nanofibrils, concentrating viral particles10.1016/j.actbio.2017.10.009 ↗
24. Vectofusin-1, a new viral entry enhancer, strongly promotes lentiviral transduction of Human hematopoietic stem cells10.1038/mtna.2013.17 ↗
25. Constraints on Human CD34+ cell fate due to lentiviral vectors can Be relieved by valproic acid10.1089/hum.2019.009 ↗
26. Inhibition of human B-cell development into plasmablasts by histone deacetylase inhibitor valproic acid10.1016/j.jaci.2013.01.018 ↗
27. Rapamycin relieves lentiviral vector transduction resistance in human and mouse hematopoietic stem cells10.1182/blood-2013-12-546218 ↗
28. A third-generation lentivirus vector with a conditional packaging system10.1128/JVI.72.11.8463-8471.1998 ↗
29. Optimized transgene delivery using third-generation lentiviruses10.1002/cpmb.125 ↗
30. Systematic comparison of 2A peptides for cloning multi-genes in a polycistronic vector10.1038/s41598-017-02460-2 ↗
31. Active immunoprophylaxis with a synthetic DNA-encoded monoclonal anti-respiratory syncytial virus scFv-fc fusion protein confers protection against infection and durable activity10.1080/21645515.2020.1748979 ↗
32. River · NODSCID Mouse Clin. Pathol. Data · 2011
33. Engineering of primary Human B cells with CRISPR/Cas9 targeted nuclease10.1038/s41598-018-30358-0 ↗
34. Reprogramming the antigen specificity of B cells using genome-editing technologies10.7554/eLife.42995 ↗
35. In vivo engineered B cells secrete high titers of broadly neutralizing anti-HIV antibodies in mice10.1038/s41587-022-01328-9 ↗
36. B cells engineered to express pathogen-specific antibodies protect against infection10.1126/sciimmunol.aax0644 ↗
37. Lentiviruses as gene transfer agents for delivery to non-dividing cells10.1016/S0958-1669(98)80029-3 ↗
38. FcRn: the neonatal fc receptor comes of age10.1038/nri2155 ↗
39. CD19 and CD20 targeted vectors induce minimal activation of resting B lymphocytes10.1371/journal.pone.0079047 ↗
40. Establishment of humanized mice from peripheral blood mononuclear cells or cord blood CD34+ hematopoietic stem cells for immune-oncology studies evaluating new therapeutic agents10.1002/cpph.77 ↗
41. Humanized mice are precious tools for preclinical evaluation of CAR T and CAR NK cell therapies10.3390/cancers12071915 ↗
42. Preclinical evaluation of B7-H3–specific chimeric antigen receptor T cells for the treatment of acute myeloid leukemia10.1158/1078-0432.CCR-20-2540 ↗
43. Bispecific binder redirected lentiviral vector enables in vivo engineering of CAR-T cells10.1136/jitc-2021-002737 ↗
44. Inactive ingredients database download, (2026).
45. Valproic acid
46. Dissecting the mechanism of histone deacetylase inhibitors to enhance the activity of zinc finger nucleases delivered by integrase-defective lentiviral vectors10.1089/hum.2013.211 ↗
47. Clinically-relevant rapamycin treatment regimens enhance CD8+ effector memory T cell function In the skin and allow their infiltration into cutaneous squamous cell carcinoma10.1080/2162402X.2018.1479627 ↗
48. Pharmacokinetic analysis of intermittent rapamycin administration in early-stage Alzheimer's Disease10.1007/s11357-025-01911-3 ↗
49. The single dose poloxamer 407 model of hyperlipidemia; systemic effects on lipids assessed using pharmacokinetic methods, and its effects on adipokines10.18433/J37G7M ↗