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