Viral-mediated gene therapy in pediatric neurological disorders

Peng, Jing; Zou, Weiwei; Wang, Xiaolei; Zhang, Zhiguo; Huo, Ran; Yang, Li

doi:10.1007/s12519-022-00669-4

Cited by 4 publications

(4 citation statements)

References 139 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…A variety of viral species have been adopted as virotherapy agents, such as Adenoviruses, retroviruses, lentiviruses, and herpes simplex viruses (Figure 4). [118][119][120][121] It has been reported that virotherapy combined with chemotherapy is a potential therapeutic modality for the improvement of transfection efficiency and therapeutic efficacy for ovarian cancer. 122,123 Zhang et al demonstrated a synergistic effect in the combination of a novel surviving promoter-based conditionally replicating adenovirus and cisplatin in the enhancement of the antitumor efficacy in ovarian cancer while Franke et al reported that tobacco mosaic virus delivered cisplatin restored efficacy in platinum-resistant ovarian cancer cells.…”

Section: Codelivery Of Viral Gene and Anticancer Drugmentioning

confidence: 99%

Nanoparticle-Based Combination Therapy for Ovarian Cancer

Wu¹,

Yang²,

Lv³

et al. 2023

IJN

View full text Add to dashboard Cite

Ovarian cancer is one of the most common malignant tumors in gynecology with a high incidence. Combination therapy, eg, administration of paclitaxel followed by a platinum anticancer drug is recommended to treat ovarian cancer due to its advantages in, eg, reducing side effects and reversing (multi)drug-resistance compared to single treatment. However, the benefits of combination therapy are often compromised. In chemo and chemo/gene combinations, co-deposition of the combined therapeutics in the tumor cells is required, which is difficult to achieve due to dramatic pharmacokinetic differences between combinational agents in free forms. Moreover, some undesired properties such as the low-water solubility of chemodrugs and the difficulty of cellular internalization of gene therapeutics also hinder the therapeutic potential. Delivery of dual or multiple agents by nanoparticles provides opportunities to tackle these limits. Nanoparticles encapsulate hydrophobic drug(s) to yield aqueous dispersions facilitating its administration and/or to accommodate hydrophilic genes facilitating its access to cells. Moreover, nanoparticle-based therapeutics can not only improve drug properties (eg, in vivo stability) and ensure the same drug disposition behavior with controlled drug ratios but also can minimize drug exposure of the normal tissues and increase drug co-accumulation at targeted tissues via passive and/or active targeting strategies. Herein, this work summarizes nanoparticle-based combination therapies, mainly including anticancer drug-based combinations and chemo/gene combinations, and emphasizes the advantageous outcomes of nanocarriers in the combination treatment of ovarian cancer. In addition, we also review mechanisms of synergetic effects resulting from different combinations.

show abstract

Section: Codelivery Of Viral Gene and Anticancer Drugmentioning

confidence: 99%

Nanoparticle-Based Combination Therapy for Ovarian Cancer

Wu¹,

Yang²,

Lv³

et al. 2023

IJN

View full text Add to dashboard Cite

show abstract

“…Currently, the commonly used adeno-associated viruses (AAVs) and lentiviral vectors show great application potential in gene delivery . Considering that an all-in-one expression cassette of TP53, PTEN, and BIM will harbor a long gene size, AAV vectors (∼4.5 kb) and lentiviral vectors (∼8 kb) show the size limitations . These vectors usually deliver one or two genes for therapeutic applications .…”

Section: Introductionmentioning

confidence: 99%

“…36 Considering that an all-in-one expression cassette of TP53, PTEN, and BIM will harbor a long gene size, AAV vectors (∼4.5 kb) and lentiviral vectors (∼8 kb) show the size limitations. 37 These vectors usually deliver one or two genes for therapeutic applications. 38 The load of an additional gene expression cassette might increase virus structure instability and reduce the efficiency of multigene coexpression.…”

Section: Introductionmentioning

confidence: 99%

Coexpression of TP53, BIM, and PTEN Enhances the Therapeutic Efficacy of Non-Small-Cell Lung Cancer

Lu,

Ma,

2024

Biomacromolecules

View full text Add to dashboard Cite

For non-small-cell lung cancer (NSCLC), the ubiquitous occurrence of concurrent multiple genomic alterations poses challenges to single-gene therapy. To increase therapeutic efficacy, we used the branch-PCR method to develop a multigene nanovector, NP-TP53-BIM-PTEN, that carried three therapeutic gene expression cassettes for coexpression. NP-TP53-BIM-PTEN exhibited a uniform size of 104.8 ± 24.2 nm and high serum stability. In cell transfection tests, NP-TP53-BIM-PTEN could coexpress TP53, BIM, and PTEN in NCI-H1299 cells and induce cell apoptosis with a ratio of up to 94.9%. Furthermore, NP-TP53-BIM-PTEN also inhibited cell proliferation with a ratio of up to 42%. In a mouse model bearing an NCI-H1299 xenograft tumor, NP-TP53-BIM-PTEN exhibited a stronger inhibitory effect on the NCI-H1299 xenograft tumor than the other test vectors without any detectable side effects. These results exhibited the potential of NP-TP53-BIM-PTEN as an effective and safe multigene nanovector to enhance NSCLC therapy efficacy, which will provide a framework for genome therapy with multigene combinations.

show abstract

“…Several drugs using AAV vectors, such as Luxturna, Zolgensma, and Hemgenix, have been approved by the FDA [ 2 , 19 , 28 ]. Moreover, several follow-up clinical trials have shown that AAV vectors are safer than other viral vectors, such as adenovirus vectors [ 20 ]. In addition, AAV vectors are widely used in research for antigen/antibody gene delivery in vivo against viral infections, such as human immunodeficiency virus, hepatitis B virus, hepatitis C virus, Nipah virus, Hendra virus, influenza A virus subtype H1N1, and dengue virus [ 4 , 6 , 18 , 21 ].…”

mentioning

confidence: 99%

Potent immunogenicity and neutralization of recombinant adeno-associated virus expressing the glycoprotein of severe fever with thrombocytopenia virus

SHIMOYAMA,

OBA,

TAKEMAE

et al. 2024

The Journal of Veterinary Medical Science

View full text Add to dashboard Cite

Severe fever with thrombocytopenia syndrome (SFTS) is an infectious disease caused by a tick-borne virus called severe fever with thrombocytopenia syndrome virus (SFTSV). In recent years, human infections through contact with ticks and through contact with the bodily fluids of infected dogs and cats have been reported; however, no vaccine is currently available. SFTSV has two glycoproteins (Gn and Gc) on its envelope, which are vaccine-target antigens involved in immunogenicity. In the present study, we constructed novel SFTS vaccine candidates using an adeno-associated virus (AAV) vector to transport the SFTSV glycoprotein genome. AAV vectors are widely used in gene therapy and their safety has been confirmed in clinical trials. Recently, AAV vectors have been used to develop influenza and SARS-CoV-2 vaccines. Two types of vaccines (AAV9-SFTSV Gn and AAV9-SFTSV Gc) carrying SFTSV Gn and Gc genes were produced. The expression of Gn and Gc proteins in HEK293T cells was confirmed by infection with vaccines. These vaccines were inoculated into mice, and the collected sera produced anti-SFTS antibodies. Furthermore, sera from AAV9-SFTSV Gn infected mice showed a potent neutralizing ability, similar to previously reported SFTS vaccine candidates that protected animals from SFTSV infection. These findings suggest that this vaccine is a promising candidate for a new SFTS vaccine.

show abstract

Viral-mediated gene therapy in pediatric neurological disorders

Cited by 4 publications

References 139 publications

Nanoparticle-Based Combination Therapy for Ovarian Cancer

Nanoparticle-Based Combination Therapy for Ovarian Cancer

Coexpression of TP53, BIM, and PTEN Enhances the Therapeutic Efficacy of Non-Small-Cell Lung Cancer

Potent immunogenicity and neutralization of recombinant adeno-associated virus expressing the glycoprotein of severe fever with thrombocytopenia virus

Contact Info

Product

Resources

About