CRISPR/Cas9 therapeutics: progress and prospects

T Li, Y Yang, H Qi, W Cui, L Zhang, X Fu, X He… - Signal transduction and …, 2023‏ - nature.com
Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated
protein 9 (Cas9) gene-editing technology is the ideal tool of the future for treating diseases …

Drug delivery systems for RNA therapeutics

K Paunovska, D Loughrey, JE Dahlman - Nature Reviews Genetics, 2022‏ - nature.com
RNA-based gene therapy requires therapeutic RNA to function inside target cells without
eliciting unwanted immune responses. RNA can be ferried into cells using non-viral drug …

CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease

A Sharma, JJ Boelens, M Cancio… - … England Journal of …, 2023‏ - Mass Medical Soc
Background Sickle cell disease is caused by a defect in the β-globin subunit of adult
hemoglobin. Sickle hemoglobin polymerizes under hypoxic conditions, producing deformed …

From target discovery to clinical drug development with human genetics

K Trajanoska, C Bhérer, D Taliun, S Zhou, JB Richards… - Nature, 2023‏ - nature.com
The substantial investments in human genetics and genomics made over the past three
decades were anticipated to result in many innovative therapies. Here we investigate the …

Sickle cell disease: a review

PL Kavanagh, TA Fasipe, T Wun - Jama, 2022‏ - jamanetwork.com
Importance Sickle cell disease (SCD) is an inherited disorder of hemoglobin, characterized
by formation of long chains of hemoglobin when deoxygenated within capillary beds …

Drug delivery systems for CRISPR-based genome editors

V Madigan, F Zhang, JE Dahlman - Nature Reviews Drug Discovery, 2023‏ - nature.com
CRISPR-based drugs can theoretically manipulate any genetic target. In practice, however,
these drugs must enter the desired cell without eliciting an unwanted immune response, so …

Mechanism and applications of CRISPR/Cas-9-mediated genome editing

M Asmamaw, B Zawdie - Biologics: targets and therapy, 2021‏ - Taylor & Francis
Clustered regularly interspaced short palindromic repeat (CRISPR) and their associated
protein (Cas-9) is the most effective, efficient, and accurate method of genome editing tool in …

[HTML][HTML] Biologic and clinical efficacy of LentiGlobin for sickle cell disease

J Kanter, MC Walters, L Krishnamurti… - … England Journal of …, 2022‏ - Mass Medical Soc
Background Sickle cell disease is characterized by the painful recurrence of vaso-occlusive
events. Gene therapy with the use of LentiGlobin for sickle cell disease (bb1111; …

Ex vivo prime editing of patient haematopoietic stem cells rescues sickle-cell disease phenotypes after engraftment in mice

KA Everette, GA Newby, RM Levine… - Nature Biomedical …, 2023‏ - nature.com
Sickle-cell disease (SCD) is caused by an A· T-to-T· A transversion mutation in the β-globin
gene (HBB). Here we show that prime editing can correct the SCD allele (HBB S) to wild …

Gene therapy: Comprehensive overview and therapeutic applications

N Sayed, P Allawadhi, A Khurana, V Singh, U Navik… - Life sciences, 2022‏ - Elsevier
Gene therapy is the product of man's quest to eliminate diseases. Gene therapy has three
facets namely, gene silencing using siRNA, shRNA and miRNA, gene replacement where …