Gene therapy clinical trials worldwide to 2017: An update

SL Ginn, AK Amaya, IE Alexander… - The journal of gene …, 2018 - Wiley Online Library
To date, almost 2600 gene therapy clinical trials have been completed, are ongoing or have
been approved worldwide. Our database brings together global information on gene therapy …

Targeted DNA insertion in plants

OX Dong, PC Ronald - … of the National Academy of Sciences, 2021 - National Acad Sciences
Conventional methods of DNA sequence insertion into plants, using Agrobacterium-
mediated transformation or microprojectile bombardment, result in the integration of the DNA …

Treating immunodeficiency through HSC gene therapy

C Booth, HB Gaspar, AJ Thrasher - Trends in molecular medicine, 2016 - cell.com
Haematopoietic stem cell (HSC) gene therapy has been successfully employed as a
therapeutic option to treat specific inherited immune deficiencies, including severe …

One small edit for humans, one giant edit for humankind? Points and questions to consider for a responsible way forward for gene editing in humans

HC Howard, CG van El, F Forzano… - European Journal of …, 2018 - nature.com
Gene editing, which allows for specific location (s) in the genome to be targeted and altered
by deleting, adding or substituting nucleotides, is currently the subject of important academic …

Indigenous perspectives and gene editing in Aotearoa New Zealand

M Hudson, ATP Mead, D Chagné… - … in bioengineering and …, 2019 - frontiersin.org
Gene editing is arguably the most significant recent addition to the modern biotechnology
toolbox, bringing both profoundly challenging and enabling opportunities. From a technical …

Variability in genome editing outcomes: challenges for research reproducibility and clinical safety

L Teboul, Y Herault, S Wells, W Qasim, G Pavlovic - Molecular Therapy, 2020 - cell.com
Genome editing tools have already revolutionized biomedical research and are also
expected to have an important impact in the clinic. However, their extensive use in research …

Invited review: small GTPases and their GAPs

AK Mishra, DG Lambright - Biopolymers, 2016 - Wiley Online Library
Widespread utilization of small GTPases as major regulatory hubs in many different
biological systems derives from a conserved conformational switch mechanism that …

The power of zebrafish in personalised medicine

S Baxendale, F van Eeden, R Wilkinson - Personalised Medicine: Lessons …, 2017 - Springer
The goal of personalised medicine is to develop tailor-made therapies for patients in whom
currently available therapeutics fail. This approach requires correlating individual patient …

Form and function of fungal and oomycete effectors

WC Sharpee, RA Dean - Fungal Biology Reviews, 2016 - Elsevier
Plants are able to recognize conserved features of potential microbial invaders and mount
an active defense in most cases. Over the course of evolution, a number of these microbes …

X‐linked neuronal migration disorders: Gender differences and insights for genetic screening

J Edey, P Soleimani‐Nouri… - International Journal …, 2023 - Wiley Online Library
Cortical development depends on neuronal migration of both excitatory and inhibitory
interneurons. Neuronal migration disorders (NMDs) are conditions characterised by …