Delivery of CRISPR-Cas tools for in vivo genome editing therapy: Trends and challenges

EA Taha, J Lee, A Hotta - Journal of Controlled Release, 2022 - Elsevier
The discovery of clustered regularly interspaced short palindromic repeats (CRISPR)
genome editing technology opened the door to provide a versatile approach for treating …

Ex vivo cell-based CRISPR/Cas9 genome editing for therapeutic applications

Y Li, Z Glass, M Huang, ZY Chen, Q Xu - Biomaterials, 2020 - Elsevier
The recently developed CRISPR/Cas9 technology has revolutionized the genome
engineering field. Since 2016, increasing number of studies regarding CRISPR therapeutics …

Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivo

R Ibraheim, PWL Tai, A Mir, N Javeed, J Wang… - Nature …, 2021 - nature.com
Adeno-associated virus (AAV) vectors are important delivery platforms for therapeutic
genome editing but are severely constrained by cargo limits. Simultaneous delivery of …

Viral vectors and extracellular vesicles: innate delivery systems utilized in CRISPR/Cas-mediated cancer therapy

SE Ahmadi, M Soleymani, F Shahriyary… - Cancer Gene …, 2023 - nature.com
Gene editing-based therapeutic strategies grant the power to override cell machinery and
alter faulty genes contributing to disease development like cancer. Nowadays, the principal …

In vivo delivery of CRISPR-Cas9 genome editing components for therapeutic applications

K Huang, D Zapata, Y Tang, Y Teng, Y Li - Biomaterials, 2022 - Elsevier
Since its mechanism discovery in 2012 and the first application for mammalian genome
editing in 2013, CRISPR-Cas9 has revolutionized the genome engineering field and created …

Hematopoietic stem cell gene-addition/editing therapy in sickle cell disease

P Germino-Watnick, M Hinds, A Le, R Chu, X Liu… - Cells, 2022 - mdpi.com
Autologous hematopoietic stem cell (HSC)-targeted gene therapy provides a one-time cure
for various genetic diseases including sickle cell disease (SCD) and β-thalassemia. SCD is …

Delivery approaches for therapeutic genome editing and challenges

I Ates, T Rathbone, C Stuart, PH Bridges, RN Cottle - Genes, 2020 - mdpi.com
Impressive therapeutic advances have been possible through the advent of zinc-finger
nucleases and transcription activator-like effector nucleases. However, discovery of the …

CRISPR technologies for stem cell engineering and regenerative medicine

MN Hsu, YH Chang, VA Truong, PL Lai… - Biotechnology …, 2019 - Elsevier
CRISPR/Cas9 system exploits the concerted action of Cas9 nuclease and programmable
single guide RNA (sgRNA), and has been widely used for genome editing. The Cas9 …

Gene therapy approaches for the treatment of hemophilia B

AB Soroka, SG Feoktistova, ON Mityaeva… - International Journal of …, 2023 - mdpi.com
In contrast to the standard enzyme-replacement therapy, administered from once per 7–14
days to 2–3 times a week in patients with severe hemophilia B, as a result of a single …

CRISPR/Cas9-mediated in vivo gene targeting corrects hemostasis in newborn and adult factor IX–knockout mice

L Wang, Y Yang, CA Breton, J White… - Blood, The Journal …, 2019 - ashpublications.org
Many genetic diseases, including hemophilia, require long-term therapeutic effects. Despite
the initial success of liver-directed adeno-associated virus (AAV) gene therapy for …