Graphite Bio: gene editing blood stem cells for sickle cell disease

2021 ◽  
Author(s):  
Michael Eisenstein
Keyword(s):  
Stem Cells ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Gene Editing ◽  
Cell Disease ◽  
Blood Stem Cells

A Highly Sensitive Assessment Tool for the Gene Editing Efficiency of Sickle Cell Disease Hemoglobin Beta Gene

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 5557-5557
Author(s):  
Mandula Borjigin ◽  
Eric Brian Kmiec ◽  
Rigumula Wu
Keyword(s):  
Stem Cells ◽  
Sickle Cell Disease ◽  
Point Mutation ◽  
Sickle Cell ◽  
Gel Electrophoresis ◽  
Gene Editing ◽  
Cell Disease ◽  
Transcription Activator ◽  
Hematopoietic Stem ◽  
Editing Efficiency

Abstract In sickle cell disease, a single point mutation in hemoglobin β gene (HBB) results in the substitution of valine for glutamic acid at position 6 of the β globin protein sequence, causing the deformation of red blood cells into a sickle (or crescent) shape. With the development of powerful gene editing tools, scientists are initiating the correction of the point mutation of HBB gene in CD34+ hematopoietic stem cells and induced pluripotent stem cells. Although the results are very exciting, the evaluation method of the gene editing is primitive. Currently, the modification at the mutation site is identified and quantified using Restriction Fragment Length Polymorphism (RFLP), which involves PCR amplification, restriction enzyme digestion and gel electrophoresis. The accuracy of the gene editing efficiency depends heavily on the quantification of the DNA bands in the gel images, which is inherently imprecise. We have developed a novel technique to quantify the correction efficiency of HBB gene editing using a fluorescence tagging of the edited DNA sequence. This method provides excellent sensitivity and accuracy, and saves time and labor, eliminating a process of gel electrophoresis. We demonstrate the assessment of gene editing in HBB of K562 cells, in which the wild type HBB (βA gene) is converted to mutant βs using the gene editing tools (i.e. Transcription Activator-Like Effector Nucleases (TALENs) and single-stranded oligo deoxynucleotides (ssODNs)). We present limited information here due to the sensitivity of the intellectual property, but will discuss in detail the experimental procedures and data at the American Society of Hematology meeting. Disclosures No relevant conflicts of interest to declare.

Reduction of BCL11A in hematopoietic stem cells through gene editing: new strategy to ameliorate the severe β-globin disorders sickle cell disease

2019 ◽  
Vol 64 (21) ◽  
pp. 1562-1564
Author(s):  
Weiqi Hong ◽  
Mengyuan Huang ◽  
Yuquan Wei ◽  
Xiawei Wei
Keyword(s):  
Stem Cells ◽  
Sickle Cell Disease ◽  
Hematopoietic Stem Cells ◽  
Sickle Cell ◽  
Gene Editing ◽  
Cell Disease ◽  
Hematopoietic Stem ◽  
New Strategy

scholarly journals A Universal Approach to Correct Various HBB Gene Mutations in Human Stem Cells for Gene Therapy of Beta-Thalassemia and Sickle Cell Disease

2017 ◽  
Vol 7 (1) ◽  
pp. 87-97 ◽  
Author(s):  
Liuhong Cai ◽  
Hao Bai ◽  
Vasiliki Mahairaki ◽  
Yongxing Gao ◽  
Chaoxia He ◽  
...  
Keyword(s):  
Stem Cells ◽  
Gene Therapy ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Gene Mutations ◽  
Beta Thalassemia ◽  
Human Stem Cells ◽  
Cell Disease ◽  
Universal Approach

scholarly journals 343. Repair of the Sickle-Cell Disease Mutation in Human Stem Cells Using CRISPR/Cas9 and Single-Stranded Oligodeoxynucleotides

2015 ◽  
Vol 23 ◽  
pp. S137
Author(s):  
Jorge Mansilla-Soto ◽  
Yan Leifman ◽  
Michel Sadelain
Keyword(s):  
Stem Cells ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Human Stem Cells ◽  
Cell Disease ◽  
Disease Mutation

CRISPR gene editing for sickle cell disease: one disease, differentiated approaches

2021 ◽  
Vol 7 (7) ◽  
pp. 851-856
Author(s):  
Lisa A Michaels, MD ◽  
Bruce E Eaton, PhD
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Gene Editing ◽  
Cell Disease

CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia

2021 ◽  
Vol 76 (6) ◽  
pp. 327-329
Author(s):  
Haydar Frangoul ◽  
David Altshuler ◽  
M. Domenica Cappellini ◽  
Yi-Shan Chen ◽  
Jennifer Domm ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Gene Editing ◽  
Cell Disease

Improving the justice‐based argument for conducting human gene editing research to cure sickle cell disease

Bioethics ◽  
2019 ◽  
Vol 34 (2) ◽  
pp. 200-202
Author(s):  
Berman Chan
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Gene Editing ◽  
Human Gene ◽  
Cell Disease
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