Non-viral Vectors in Gene Therapy: Recent Development, Challenges, and Prospects

Zu, Hui; Gao, Danchen

doi:10.1208/s12248-021-00608-7

Cited by 243 publications

(193 citation statements)

References 85 publications

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“…Viral vectors [ 91 ], which are highly effective due to their total or partial ability to integrate into the genome, are prone to a high rate of adverse events. Conversely, non-viral vectors are extremely safe but not as effective as they rarely allow for therapeutic levels of the expressed protein to be achieved [ 92 , 93 ]. One could also classify cell therapy protocols as a function of the target cells used.…”

Section: Discussionmentioning

confidence: 99%

High Mutational Heterogeneity, and New Mutations in the Human Coagulation Factor V Gene. Future Perspectives for Factor V Deficiency Using Recombinant and Advanced Therapies

Bernal

Peláez

Alías

et al. 2021

IJMS

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Factor V is an essential clotting factor that plays a key role in the blood coagulation cascade on account of its procoagulant and anticoagulant activity. Eighty percent of circulating factor V is produced in the liver and the remaining 20% originates in the α-granules of platelets. In humans, the factor V gene is about 80 kb in size; it is located on chromosome 1q24.2, and its cDNA is 6914 bp in length. Furthermore, nearly 190 mutations have been reported in the gene. Factor V deficiency is an autosomal recessive coagulation disorder associated with mutations in the factor V gene. This hereditary coagulation disorder is clinically characterized by a heterogeneous spectrum of hemorrhagic manifestations ranging from mucosal or soft-tissue bleeds to potentially fatal hemorrhages. Current treatment of this condition consists in the administration of fresh frozen plasma and platelet concentrates. This article describes the cases of two patients with severe factor V deficiency, and of their parents. A high level of mutational heterogeneity of factor V gene was identified, nonsense mutations, frameshift mutations, missense changes, synonymous sequence variants and intronic changes. These findings prompted the identification of a new mutation in the human factor V gene, designated as Jaén-1, which is capable of altering the procoagulant function of factor V. In addition, an update is provided on the prospects for the treatment of factor V deficiency on the basis of yet-to-be-developed recombinant products or advanced gene and cell therapies that could potentially correct this hereditary disorder.

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Section: Discussionmentioning

confidence: 99%

High Mutational Heterogeneity, and New Mutations in the Human Coagulation Factor V Gene. Future Perspectives for Factor V Deficiency Using Recombinant and Advanced Therapies

Bernal

Peláez

Alías

et al. 2021

IJMS

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“…A careful consideration of the impact on the use of vectors for vaccine production and the plausible negative impact in the development of suitable gene therapy medicinal products, should be on top of the scientific and regulatory table discussion in order to facilitate access to patients suffering monogenic diseases and also prevent unnecessary delays in future vector vaccines development. Hopefully, this issue will become less relevant when new generations of non-viral vectors will start to show efficacy in the treatment of monogenic diseases [ 8 ]. As in many other aspects, this pandemic is challenging our capacity to coordinate, plan ahead and align different medical objectives.…”

Section: Main Textmentioning

confidence: 99%

Gene therapies and COVID-19 vaccines: a necessary discussion in relation with viral vector-based approaches

Aledo‐Serrano

Gil‐Nagel

Isla³

et al. 2021

Orphanet J Rare Dis

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The COVID-19 pandemic is adding an unanticipated concern for those affected by genetic diseases. Most of the new treatment achievements for these patients are made possible as a result of advances in viral-based products. Among them, adenoviruses (AdV) and especially adeno-associated viruses (AAV) are important players. The concerns and the conversation around this issue have increased as COVID-19 vaccines approach the market. What if the viral vectors become the mainstream strategy for vaccine development? Will the immune response elicited against the vector compromise the efficacy of future gene therapies? Patients with genetic diseases and patient advocacy groups are requesting information to the medical community about the potential impact of these vaccines in future gene therapy treatments, and physicians and scientists are not able to provide satisfactory answer yet. Importantly, the frequency of cross-reactivity among different AAV serotypes can be as high as 50%. This would have potential implications for patients with genetic disorders who could benefit from gene therapies, often coming in the form of AAV-based gene therapies. As in many other aspects, this pandemic is challenging our capacity to coordinate, plan ahead and align different medical objectives. In this case, having such conversation early on might allow us to make the right choices while we are still on time.

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“…36 Overall, other than the more than twenty gene therapy products approved, 37 clinical evaluations of NVVs for other diseases continue to increase in number and scope, including polymer-based NVVs (Table 4). [38][39][40] This suggests that they will form a vital component of future skin disease treatments. This review will focus on polymer-based NVVs for gene delivery in skin disease treatment.…”

Section: Introductionmentioning

confidence: 99%

Polymer-based non-viral vectors for gene therapy in the skin

Tortajada¹,

Felip²,

Vicent

2022

Polym. Chem.

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Non-viral Vectors in Gene Therapy: Recent Development, Challenges, and Prospects

Cited by 243 publications

References 85 publications

High Mutational Heterogeneity, and New Mutations in the Human Coagulation Factor V Gene. Future Perspectives for Factor V Deficiency Using Recombinant and Advanced Therapies

High Mutational Heterogeneity, and New Mutations in the Human Coagulation Factor V Gene. Future Perspectives for Factor V Deficiency Using Recombinant and Advanced Therapies

Gene therapies and COVID-19 vaccines: a necessary discussion in relation with viral vector-based approaches

Polymer-based non-viral vectors for gene therapy in the skin

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