# Designing Lentiviral Vectors for Gene Therapy of Genetic Diseases

## TL;DR

This paper reviews how lentiviral vectors are designed to treat genetic diseases by controlling gene expression in clinical gene therapy.

## Contribution

The paper provides a focused review on the design of lentiviral vectors for gene therapy, emphasizing clinical development and gene expression control.

## Key findings

- Lentiviral vectors are widely used in gene therapy for monogenic diseases.
- Ex vivo gene transfer using these vectors is common in clinical applications.
- Transcriptional and post-transcriptional control mechanisms are crucial for effective gene therapy.

## Abstract

Lentiviral vectors are the most frequently used tool to stably transfer and express genes in the context of gene therapy for monogenic diseases. The vast majority of clinical applications involves an ex vivo modality whereby lentiviral vectors are used to transduce autologous somatic cells, obtained from patients and re-delivered to patients after transduction. Examples are hematopoietic stem cells used in gene therapy for hematological or neurometabolic diseases or T cells for immunotherapy of cancer. We review the design and use of lentiviral vectors in gene therapy of monogenic diseases, with a focus on controlling gene expression by transcriptional or post-transcriptional mechanisms in the context of vectors that have already entered a clinical development phase.