Microfluidic production of nanogels as alternative triple transfection reagents for the manufacture of adeno-associated virus vectors

Whiteley, Zoe; Massaro, Giulia; Gkogkos, Georgios; Gavriilidis, Asterios; Waddington, Simon; Rahim, Ahad A.; Craig, Duncan Q.M.

doi:10.1039/d2nr06401d

Cited by 3 publications

(2 citation statements)

References 52 publications

(74 reference statements)

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“…Chemical transient transfection is popular due to simplicity and availability of raw materials but has limited yield per cell and requires good manufacturing process (GMP) grade plasmids [83–85]. New technology has emerged to scale transient transfections, including packed bed reactors [86–88], conversion to suspension HEK293 lines [89], AAV process intensification [90 ▪ ,91 ▪ ] and the use of novel chemical additives as transfection reagents [84,89,92–94,95 ▪ ,96 ▪ ]. Nonplasmid DNA sources such as doggybone DNA and novel transfection reagents could facilitate manufacturing significantly, along with the use of certain chemical additives to boost rAAV production [95 ▪ ,97–99].…”

Section: Challenges and Potential Solutions In Gene Therapymentioning

confidence: 99%

Current avenues of gene therapy in Pompe disease

Leon-Astudillo,

Trivedi,

Sun

et al. 2023

Current Opinion in Neurology

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Purpose of review Pompe disease is a rare, inherited, devastating condition that causes progressive weakness, cardiomyopathy and neuromotor disease due to the accumulation of glycogen in striated and smooth muscle, as well as neurons. While enzyme replacement therapy has dramatically changed the outcome of patients with the disease, this strategy has several limitations. Gene therapy in Pompe disease constitutes an attractive approach due to the multisystem aspects of the disease and need to address the central nervous system manifestations. This review highlights the recent work in this field, including methods, progress, shortcomings, and future directions. Recent findings Recombinant adeno-associated virus (rAAV) and lentiviral vectors (LV) are well studied platforms for gene therapy in Pompe disease. These products can be further adapted for safe and efficient administration with concomitant immunosuppression, with the modification of specific receptors or codon optimization. rAAV has been studied in multiple clinical trials demonstrating safety and tolerability. Summary Gene therapy for the treatment of patients with Pompe disease is feasible and offers an opportunity to fully correct the principal pathology leading to cellular glycogen accumulation. Further work is needed to overcome the limitations related to vector production, immunologic reactions and redosing.

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Section: Challenges and Potential Solutions In Gene Therapymentioning

confidence: 99%

Current avenues of gene therapy in Pompe disease

Leon-Astudillo,

Trivedi,

Sun

et al. 2023

Current Opinion in Neurology

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“…These channels enable the precise manipulation and control of uids, facilitating rapid mixing and minimizing sample volumes. Microuidics has been extensively used to generate polymer nanogels from polysaccharides such as sodium alginate, 6,7 chitosan, 8,9 hyaluronic acid, 10 etc. These are typically produced by either the ionic crosslinking of the charged polymers with an oppositely charged species or by precipitation in a non-solvent.…”

Section: Introductionmentioning

confidence: 99%

One-step flow synthesis of size-controlled polymer nanogels in a fluorocarbon microfluidic chip

Montalbo,

Wu,

2024

RSC Adv.

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Nanogels: Recent Advances in Synthesis and Biomedical Applications

Mastella,

Todaro,

Luin

2024

Nanomaterials

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In the context of advanced nanomaterials research, nanogels (NGs) have recently gained broad attention for their versatility and promising biomedical applications. To date, a significant number of NGs have been developed to meet the growing demands in various fields of biomedical research. Summarizing preparation methods, physicochemical and biological properties, and recent applications of NGs may be useful to help explore new directions for their development. This article presents a comprehensive overview of the latest NG synthesis methodologies, highlighting advances in formulation with different types of hydrophilic or amphiphilic polymers. It also underlines recent biomedical applications of NGs in drug delivery and imaging, with a short section dedicated to biosafety considerations of these innovative nanomaterials. In conclusion, this article summarizes recent innovations in NG synthesis and their numerous applications, highlighting their considerable potential in the biomedical field.

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Microfluidic production of nanogels as alternative triple transfection reagents for the manufacture of adeno-associated virus vectors

Abstract: Microfluidic produced nanogels provide an alternative to standard transfection reagents for the manufacture of adeno-associated viral vectors (AAVs), with equivelant viral titres demonstrated.

Cited by 3 publications

References 52 publications

Current avenues of gene therapy in Pompe disease

Current avenues of gene therapy in Pompe disease

One-step flow synthesis of size-controlled polymer nanogels in a fluorocarbon microfluidic chip

Nanogels: Recent Advances in Synthesis and Biomedical Applications

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