Dataset Information

Systematic review, meta-analysis, and future scope of lentivirus-mediated gene therapy

ABSTRACT: Gene therapy has emerged as a promising therapeutic option for many inherited and acquired diseases. Lentivirus-mediated gene transfer is a powerful technique to deliver therapeutic genes into target cells and offers several advantages over other gene delivery methods. This systematic review explores the mechanistic intricacies of lentivirus-based vectors and provides the current status on their safety and efficacy in therapeutic applications underpinned by meta-analysis of clinical trials reported from across the globe. Our findings elucidate that gene therapy using lentiviruses has been extended to various diseases spanning from immunodeficiencies and hemoglobinopathies to specific malignancies and lysosomal storage diseases. Although the meta-analysis indicates a positive trend with respect to safety, the results pertaining to efficacy were not significant, highlighting the importance of continued investigation and cautious interpretation of the results. The findings from this study underscore the imperative for advancing research in vector development followed by preclinical and clinical assessments, to elevate the potential of lentiviral gene therapy as a potential therapeutic modality in the future.

ORGANISM(S): NA

SUBMITTER:

PROVIDER: S-BSST1312 | biostudies-other |

REPOSITORIES: biostudies-other

ACCESS DATA

Similar Datasets

Project description:BackgroundOsteosarcopenia, a combination of osteopenia/osteoporosis and sarcopenia, is a common condition among older adults. While numerous studies and meta-analyses have been conducted on the treatment of osteoporosis, the pharmacological treatment of osteosarcopenia still lacks evidence. Denosumab, a human monoclonal antibody, has shown encouraging results for the treatment of osteosarcopenia. Our systematic review and meta-analysis aimed to investigate the potential dual role of denosumab as an anti-resorptive agent and for other beneficial muscle-related effects in patients with osteosarcopenia, and to evaluate whether denosumab can be a treatment of choice compared to bisphosphonate.MethodsRelevant literature was collated from the Cochrane Central Register of Controlled Trials (CENTRAL), PubMed, and Google Scholar databases. The primary outcome was denosumab's effect on lumbar spine bone mineral density (LS BMD), handgrip strength, and gait speed change. The secondary outcome was the effect of denosumab on appendicular lean mass (ALM). The outcomes were presented as mean difference (MD). A random effects model was used in the analysis to represent the population. The risk of bias was assessed using funnel plots.ResultsOut of the 3,074 studies found, four full-text studies met the inclusion criteria, including 264 and 244 participants in the intervention and control groups, respectively. Regarding a primary outcome, our meta-analysis showed that denosumab showed no significant differences in LS BMD and gait speed changes compared to other agents-MD=0.37, 95% confidence interval (CI), -0.35 to 0.79; p=0.09 and MD=0.11; 95% CI, -0.18 to 0.40; p=0.46, respectively. Denosumab had a significant effect on handgrip strength change compared to standard agents-MD=5.16; 95% CI, 1.38 to 18.94; p=0.007, based on the random effects model.ConclusionsDenosumab was better than bisphosphonate and placebo in improving muscle strength (handgrip strength). Therefore, denosumab may be favored in individuals with osteosarcopenia to improve muscular performance and reduce fall risk.

Dataset Information

Systematic review, meta-analysis, and future scope of lentivirus-mediated gene therapy

Similar Datasets

OmicsDI is part of the ELIXIR infrastructure

Tweets