[HTML][HTML] Gene therapy: advances, challenges and perspectives

GAR Gonçalves, RMA Paiva - Einstein (Sao Paulo), 2017 - SciELO Brasil
Next, another Chinese group also reported the conduction of the same process done with …
any new technology, need more clarifying preclinical studies. In the future, there is the promise

Gene therapy comes of age

CE Dunbar, KA High, JK Joung, DB Kohn, K Ozawa… - Science, 2018 - science.org
discoveries leading to the development of successful gene … editing trials are expected to
open over the next decade. … clinical promise, and we will limit our discussions to these vectors. …

Clinical development of gene therapy: results and lessons from recent successes

SRP Kumar, DM Markusic, M Biswas, KA High… - Molecular therapy …, 2016 - cell.com
Therapeutic gene transfer holds the promise of providing … Advances in the development of
gene therapy vector systems, … in most patients, who were subsequently able to resolve pre-…

[HTML][HTML] The once and future gene therapy

K Bulaklak, CA Gersbach - Nature communications, 2020 - nature.com
… of therapies and laid the foundation for next-generation technologies. This comment summarizes
recent advances … Despite the promise of this approach, it does not address correction of …

Gene therapy leaves a vicious cycle

R Goswami, G Subramanian, L Silayeva… - Frontiers in …, 2019 - frontiersin.org
… used in gene therapy, advances, challenges, and perspectives. … the next 10 years, both
clinical trials and pharmaceutical industry are anticipated to benefit immensely from gene therapy

Gene therapy for neurological disorders: progress and prospects

BE Deverman, BM Ravina, KS Bankiewicz… - Nature Reviews Drug …, 2018 - nature.com
… Here, we discuss key considerations and challenges in the … advances have resulted in a
resurgence of interest in gene … substantial promise for achieving improved biological profiles for …

The promise and challenge of therapeutic genome editing

JA Doudna - Nature, 2020 - nature.com
breakthroughs in agriculture and synthetic biology 6,7,8,9 . Building on past gene therapy
… The next section describes current genome-editing technologies that offer the potential of …

Nanoparticles for cancer gene therapy: Recent advances, challenges, and strategies

K Wang, FM Kievit, M Zhang - Pharmacological research, 2016 - Elsevier
Gene therapy holds unique promise in meeting these goals by offering a means of specific
… (5-FU) that is subsequently converted by endogenous enzymes into potent pyrimidine …

Between hope and reality: treatment of genetic diseases through nucleic acid-based drugs

V Baylot, TK Le, D Taïeb, P Rocchi… - Communications …, 2024 - nature.com
… After decades of promises, tempered by great deal of failures… 25 years after the first gene
therapy attempt to treat children … the MHRA represents a major breakthrough in the field. On …

Non-viral vectors in gene therapy: recent development, challenges, and prospects

H Zu, D Gao - The AAPS journal, 2021 - Springer
Gene therapy has been experiencing a breakthrough in … The model has provided a new
tool to study the subsequent … cancer cells, which shows promise for targeted gene therapy. …