The promise and challenge of therapeutic genome editing
JA Doudna - Nature, 2020 - nature.com
Genome editing, which involves the precise manipulation of cellular DNA sequences to alter
cell fates and organism traits, has the potential to both improve our understanding of human …
cell fates and organism traits, has the potential to both improve our understanding of human …
Gene therapy using haematopoietic stem and progenitor cells
Haematopoietic stem and progenitor cell (HSPC) gene therapy has emerged as an effective
treatment modality for monogenic disorders of the blood system such as primary …
treatment modality for monogenic disorders of the blood system such as primary …
Highly efficient therapeutic gene editing of human hematopoietic stem cells
Re-expression of the paralogous γ-globin genes (HBG1/2) could be a universal strategy to
ameliorate the severe β-globin disorders sickle cell disease (SCD) and β-thalassemia by …
ameliorate the severe β-globin disorders sickle cell disease (SCD) and β-thalassemia by …
Identification of non-coding silencer elements and their regulation of gene expression
Cell type-and differentiation-specific gene expression is precisely controlled by genomic non-
coding regulatory elements (NCREs), which include promoters, enhancers, silencers and …
coding regulatory elements (NCREs), which include promoters, enhancers, silencers and …
Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease
Sickle cell disease (SCD) is the most common serious monogenic disease with 300,000
births annually worldwide. SCD is an autosomal recessive disease resulting from a single …
births annually worldwide. SCD is an autosomal recessive disease resulting from a single …
Gene editing and CRISPR in the clinic: current and future perspectives
Genome editing technologies, particularly those based on zinc-finger nucleases (ZFNs),
transcription activator-like effector nucleases (TALENs), and CRISPR (clustered regularly …
transcription activator-like effector nucleases (TALENs), and CRISPR (clustered regularly …
Comprehensive review of CRISPR-based gene editing: mechanisms, challenges, and applications in cancer therapy
M Chehelgerdi, M Chehelgerdi… - Molecular cancer, 2024 - Springer
The CRISPR system is a revolutionary genome editing tool that has the potential to
revolutionize the field of cancer research and therapy. The ability to precisely target and edit …
revolutionize the field of cancer research and therapy. The ability to precisely target and edit …
Comprehensive analysis and accurate quantification of unintended large gene modifications induced by CRISPR-Cas9 gene editing
Most genome editing analyses to date are based on quantifying small insertions and
deletions. Here, we show that CRISPR-Cas9 genome editing can induce large gene …
deletions. Here, we show that CRISPR-Cas9 genome editing can induce large gene …
Defining global strategies to improve outcomes in sickle cell disease: a Lancet Haematology Commission
Executive summary All over the world, people with sickle cell disease (an inherited
condition) have premature deaths and preventable severe chronic complications, which …
condition) have premature deaths and preventable severe chronic complications, which …
Hematopoietic stem cell gene therapy: progress and lessons learned
RA Morgan, D Gray, A Lomova, DB Kohn - Cell stem cell, 2017 - cell.com
The use of allogeneic hematopoietic stem cells (HSCs) to treat genetic blood cell diseases
has become a clinical standard but is limited by the availability of suitable matched donors …
has become a clinical standard but is limited by the availability of suitable matched donors …