[HTML][HTML] Gene therapy clinical trials, where do we go? An overview

F Arabi, V Mansouri, N Ahmadbeigi - Biomedicine & Pharmacotherapy, 2022 - Elsevier
There have been many ups and downs since the introduction of gene therapy as a
therapeutic modality for diseases. However, the journey of gene therapy has reached a …

Engineering precision nanoparticles for drug delivery

MJ Mitchell, MM Billingsley, RM Haley… - Nature reviews drug …, 2021 - nature.com
In recent years, the development of nanoparticles has expanded into a broad range of
clinical applications. Nanoparticles have been developed to overcome the limitations of free …

Successes and challenges in clinical gene therapy

DB Kohn, YY Chen, MJ Spencer - Gene therapy, 2023 - nature.com
Despite the ups and downs in the field over three decades, the science of gene therapy has
continued to advance and provide enduring treatments for increasing number of diseases …

CRISPR gene therapy: applications, limitations, and implications for the future

F Uddin, CM Rudin, T Sen - Frontiers in oncology, 2020 - frontiersin.org
A series of recent discoveries harnessing the adaptive immune system of prokaryotes to
perform targeted genome editing is having a transformative influence across the biological …

Viral vectors in gene therapy: where do we stand in 2023?

K Lundstrom - Viruses, 2023 - mdpi.com
Viral vectors have been used for a broad spectrum of gene therapy for both acute and
chronic diseases. In the context of cancer gene therapy, viral vectors expressing anti-tumor …

Organelle-targeted therapies: a comprehensive review on system design for enabling precision oncology

J Yang, A Griffin, Z Qiang, J Ren - Signal transduction and targeted …, 2022 - nature.com
Cancer is a major threat to human health. Among various treatment methods, precision
therapy has received significant attention since the inception, due to its ability to efficiently …

Defining global strategies to improve outcomes in sickle cell disease: a Lancet Haematology Commission

FB Piel, DC Rees, MR DeBaun, O Nnodu… - The Lancet …, 2023 - thelancet.com
Executive summary All over the world, people with sickle cell disease (an inherited
condition) have premature deaths and preventable severe chronic complications, which …

A review of different types of liposomes and their advancements as a form of gene therapy treatment for breast cancer

GYW Tseu, KA Kamaruzaman - Molecules, 2023 - mdpi.com
Breast cancer incidence and mortality rates have increased exponentially during the last
decade, particularly among female patients. Current therapies, including surgery and …

Delivering CRISPR: a review of the challenges and approaches

CA Lino, JC Harper, JP Carney, JA Timlin - Drug delivery, 2018 - Taylor & Francis
Gene therapy has long held promise to correct a variety of human diseases and defects.
Discovery of the Clustered Regularly-Interspaced Short Palindromic Repeats (CRISPR), the …

Gene therapy using haematopoietic stem and progenitor cells

G Ferrari, AJ Thrasher, A Aiuti - Nature Reviews Genetics, 2021 - nature.com
Haematopoietic stem and progenitor cell (HSPC) gene therapy has emerged as an effective
treatment modality for monogenic disorders of the blood system such as primary …