Recent advances in lentiviral vectors for gene therapy

X Wang, C Ma, R Rodríguez Labrada, Z Qin… - Science China Life …, 2021 - Springer
Lentiviral vectors (LVs), derived from human immunodeficiency virus, are powerful tools for
modifying the genes of eukaryotic cells such as hematopoietic stem cells and neural cells …

[HTML][HTML] Powering prescription: Mitochondria as “Living Drugs”–Definition, clinical applications, and industry advancements

A Caicedo, E Morales, A Moyano, S Peñaherrera… - Pharmacological …, 2024 - Elsevier
Mitochondria's role as engines and beacons of metabolism and determinants of cellular
health is being redefined through their therapeutic application as “Living Drugs”(LDs) …

Beyond the promise: Evaluating and mitigating off-target effects in CRISPR gene editing for safer therapeutics

R Lopes, MK Prasad - Frontiers in Bioengineering and Biotechnology, 2024 - frontiersin.org
Over the last decade, CRISPR has revolutionized drug development due to its potential to
cure genetic diseases that currently do not have any treatment. CRISPR was adapted from …

Regulatory considerations for clinical trial applications with CRISPR-based medicinal products

B Anliker, L Childs, J Rau, M Renner, S Schüle… - The CRISPR …, 2022 - liebertpub.com
Since first proposed as a new tool for gene targeting and genome editing, CRISPR
technology has quickly advanced into the clinical stage. Initial studies highlight the potential …

Reflection on the enactment and impact of safety laws for regenerative medicine in Japan

K Takashima, M Morrison, J Minari - Stem Cell Reports, 2021 - cell.com
Japan's Act on the Safety of Regenerative Medicine (ASRM) created an innovative
regulatory framework intended to safely promote the clinical development of stem cell-based …

The Dawn of In Vivo Gene Editing Era: A Revolution in the Making

SK Niazi - Biologics, 2023 - mdpi.com
Gene or genome editing (GE) revises, removes, or replaces a mutated gene at the DNA
level; it is a tool. Gene therapy (GT) offsets mutations by introducing a “normal” version of the …

Moving somatic gene editing to the clinic: Routes to market access and reimbursement in Europe

T Rigter, D Klein, SS Weinreich… - European Journal of …, 2021 - nature.com
Somatic gene editing (SGE) holds great promise for making genetic therapy possible for
many monogenic conditions very soon. Is our current system of European market …

Gene Editing: The Regulatory Perspective

SK Niazi - Encyclopedia, 2023 - mdpi.com
Definition Gene or genome editing, often known as GE, is a technique utilized to modify,
eliminate, or substitute a mutated gene at the DNA level. It serves as a valuable tool in the …

Toxicity Aspects and Ethical Issues of Bioprinting

N Al Hashimi… - 3D Bioprinting from Lab to …, 2024 - Wiley Online Library
Summary Three‐dimensional (3D) bioprinting is revolutionizing the medical industry.
Bioprinting could help overcome the limitations of organ transplant waiting times and …

[HTML][HTML] Gene therapy and its applications

A Bansal, R Prakash, S Agarwal… - Journal of Medical …, 2023 - journals.lww.com
Gene therapy is the treatment of abnormal or mutated genes present in cells through the
addition of healthy genes or replacement/deletion/site-specific modification of faulty genes …