Detalhe da pesquisa
1.
Targeted replacement of full-length CFTR in human airway stem cells by CRISPR-Cas9 for pan-mutation correction in the endogenous locus.
Mol Ther
; 30(1): 223-237, 2022 01 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-33794364
2.
Genome editing of donor-derived T-cells to generate allogenic chimeric antigen receptor-modified T cells: Optimizing αß T cell-depleted haploidentical hematopoietic stem cell transplantation.
Haematologica
; 106(3): 847-858, 2021 03 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-32241852
3.
AAV-CRISPR Gene Editing Is Negated by Pre-existing Immunity to Cas9.
Mol Ther
; 28(6): 1432-1441, 2020 06 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-32348718
4.
Highly efficient editing of the ß-globin gene in patient-derived hematopoietic stem and progenitor cells to treat sickle cell disease.
Nucleic Acids Res
; 47(15): 7955-7972, 2019 09 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-31147717
5.
A Burden of Rare Variants Associated with Extremes of Gene Expression in Human Peripheral Blood.
Am J Hum Genet
; 98(2): 299-309, 2016 Feb 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-26849112
6.
CD7-edited T cells expressing a CD7-specific CAR for the therapy of T-cell malignancies.
Blood
; 130(3): 285-296, 2017 07 20.
Artigo
em Inglês
| MEDLINE | ID: mdl-28539325
7.
CRISPR/Cas9-Based Genome Editing for Disease Modeling and Therapy: Challenges and Opportunities for Nonviral Delivery.
Chem Rev
; 117(15): 9874-9906, 2017 Aug 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-28640612
8.
Towards Device-Independent Information Processing on General Quantum Networks.
Phys Rev Lett
; 120(2): 020504, 2018 Jan 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-29376705
9.
Examination of CRISPR/Cas9 design tools and the effect of target site accessibility on Cas9 activity.
Exp Physiol
; 103(4): 456-460, 2018 04 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-28303677
10.
Genome editing for inborn errors of metabolism: advancing towards the clinic.
BMC Med
; 15(1): 43, 2017 02 27.
Artigo
em Inglês
| MEDLINE | ID: mdl-28238287
11.
The Neisseria meningitidis CRISPR-Cas9 System Enables Specific Genome Editing in Mammalian Cells.
Mol Ther
; 24(3): 645-54, 2016 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-26782639
12.
Nuclease Target Site Selection for Maximizing On-target Activity and Minimizing Off-target Effects in Genome Editing.
Mol Ther
; 24(3): 475-87, 2016 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-26750397
13.
Streptococcus thermophilus CRISPR-Cas9 Systems Enable Specific Editing of the Human Genome.
Mol Ther
; 24(3): 636-44, 2016 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-26658966
14.
Treating hemoglobinopathies using gene-correction approaches: promises and challenges.
Hum Genet
; 135(9): 993-1010, 2016 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-27314256
15.
Identification and Validation of CRISPR/Cas9 Off-Target Activity in Hematopoietic Stem and Progenitor Cells.
Methods Mol Biol
; 2429: 281-306, 2022.
Artigo
em Inglês
| MEDLINE | ID: mdl-35507169
16.
Electroporation-Mediated Delivery of Cas9 Ribonucleoproteins Results in High Levels of Gene Editing in Primary Hepatocytes.
CRISPR J
; 5(3): 397-409, 2022 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-35238624
17.
Leveraging Directed Causal Discovery to Detect Latent Common Causes in Cause-Effect Pairs.
IEEE Trans Neural Netw Learn Syst
; PP2022 Oct 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-36201419
18.
Technology readiness levels for machine learning systems.
Nat Commun
; 13(1): 6039, 2022 10 20.
Artigo
em Inglês
| MEDLINE | ID: mdl-36266298
19.
Tools for experimental and computational analyses of off-target editing by programmable nucleases.
Nat Protoc
; 16(1): 10-26, 2021 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-33288953
20.
In vivo genome editing at the albumin locus to treat methylmalonic acidemia.
Mol Ther Methods Clin Dev
; 23: 619-632, 2021 Dec 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-34901307