Ready for repair? Gene editing enters the clinic for the treatment of human disease

MPT Ernst, M Broeders, P Herrero-Hernandez… - … Methods & Clinical …, 2020 - cell.com
We present an overview of clinical trials involving gene editing using clustered interspaced
short palindromic repeats (CRISPR)-CRISPR-associated protein 9 (Cas9), transcription …

Gene editing: regulatory and translation to clinic

D Ando, K Meyer - Hematology/Oncology Clinics, 2017 - hemonc.theclinics.com
… This assessment was conducted post genome editing treatment (before cell administration
to animals or human subjects) and then blood samples were collected and evaluated at …

[PDF][PDF] A new era of gene editing for the treatment of human diseases

KC Mandip, CJ Steer - Swiss medical weekly, 2019 - smw.ch
… The CRISPR/Cas9 system with its myriad of applications has created an entirely new approach
to the study and treatment of diseases with aberrant epigenetic mechanisms. The clinical

Genome editing: a tool for research and therapy: targeted genome editing hits the clinic

A Lombardo, L Naldini - Nature medicine, 2014 - nature.com
… is thus set to become a potential game changer in gene therapy. In the HIV clinical trial 1
, T cells harvested from HIV-infected people were treated with an adenoviral vector that …

Gene editing in clinical practice: where are we?

RD Mittal - Indian Journal of Clinical Biochemistry, 2019 - Springer
… of disease process and their treatment in near future. Undoubtedly this field is
revolutionizing in current era and may open new vistas in the treatment of fatal genetic disease. …

Gene editing and CRISPR in the clinic: current and future perspectives

MP Hirakawa, R Krishnakumar, JA Timlin… - Bioscience …, 2020 - portlandpress.com
… In this review, we will first discuss the state of gene editing technologies and their use as
treatments for human disease with a specific focus on CRISPR-based therapies that are …

Gene editing on center stage

RO Bak, N Gomez-Ospina, MH Porteus - Trends in Genetics, 2018 - cell.com
genome editing using ZFNs and TALENs has already reached clinical trials, the pace at
which genome editing … using genome editing to understand, treat, and cure genetic diseases. …

Refining strategies to translate genome editing to the clinic

TI Cornu, C Mussolino, T Cathomen - Nature medicine, 2017 - nature.com
gene-editing studies have found their way into the clinic, we are still waiting for clinical proof
that gene editing is actually able to treat … -up of the first clinical gene-editing trials in CD34 + …

The potential of CRISPR/Cas9 gene editing as a treatment strategy for inherited diseases

SA Abdelnour, L Xie, AA Hassanin, E Zuo… - Frontiers in cell and …, 2021 - frontiersin.org
… Prime editing further expands the scope and capabilities of genome editing. A number of
additional studies may further advance base-editing treatments for progeria towards clinical

With first CRISPR trials, gene editing moves toward the clinic

T Hampton - Jama, 2020 - jamanetwork.com
… study will likely have wider-reaching clinical applications. … and public debate are needed as
gene editing gears up for the clinic. … “We don’t want to be developing CRISPR treatments that …