2. Progress in PID gene therapy clinical trials 2.1. ADA-SCID Under normal physiological circumstances, DNA and RNA are broken down inside the cell into toxic metabolites called deoxyadenosine and adenosine respectively, which when acted upon by the enzyme adenosine deaminase (ADA), are converted in...
Note this section should not be used to describe any competing interests. Footnotes Footnotes can be used to give additional information, which may include the citation of a reference included in the reference list. They should not consist solely of a reference citation, and they should never ...
We discuss the progress made and future challenges for gene therapy and contrast gene therapy with gene-editing strategies.This is a preview of subscription content, access via your institution Access optionsAccess Nature and 54 other Nature Portfolio journals Get Nature+, our best-value online-...
RNA therapy refers to the treatment or prevention of diseases using RNA-based molecules. The recent advent of a series of effective messenger RNA-based vaccines in response to the COVID-19 pandemic has reignited research interest in RNA therapy. Based on
However, it was important to note that this analysis was not a comparison of efficacy. While LITT may have a cost advantage, assertions could not be made at this time regarding which modality is more effective in prolonging the life of the patient. It was assumed that patients discharged ...
validate these predictions. This allows researchers to identify and avoid non-selective site sites before they cause unintended mutations [220]. Finally, it is important to note that off-target effects can also arise from the delivery method used to deliver the CRISPR machinery to the cells [221...
Template for Intervention Description and Replication MCDID: Minimum clinically important difference ODI: Oswestry Disability Index PSEQ: Pain Self-Efficacy Questionnaire GPE: Global Perceived Effect of improvement with treatment STES: Standford ExpectationTreatment Scale PHQ-9: Patient Health Questio...
Template A review should describe an overview of recent progress in important fields of research, providing an appreciation of the significance of the work, and an outlook into potential future directions. Unstructured abstract ≤ 300 words
Smart nanoparticles, which can respond to biological cues or be guided by them, are emerging as a promising drug delivery platform for precise cancer treatment. The field of oncology, nanotechnology, and biomedicine has witnessed rapid progress, leading to innovative developments in smart nanoparticles...
W. Y. Gene therapy for neurological disorders: progress and prospects. Nat. Rev. Drug Discov. 17, 641–659 (2018). This is an up-to-date review on the current trends and challenges for AAV-based CNS gene therapy. CAS PubMed Google Scholar Petit, L., Khanna, H. & Punzo, C. ...