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F. Ann Ran
Broad Institute
39Publications
10H-index
17.6kCitations
Publications 26
Newest
#1Mari Yamada (UTokyo: University of Tokyo)H-Index: 1
#2Yuto Watanabe (UTokyo: University of Tokyo)H-Index: 1
Last.Osamu Nureki (UTokyo: University of Tokyo)H-Index: 56
view all 10 authors...
Summary The RNA-guided endonuclease Cas9 generates a double-strand break at DNA target sites complementary to the guide RNA and has been harnessed for the development of a variety of new technologies, such as genome editing. Here, we report the crystal structures of Campylobacter jejuni Cas9 (CjCas9), one of the smallest Cas9 orthologs, in complex with an sgRNA and its target DNA. The structures provided insights into a minimal Cas9 scaffold and revealed the remarkable mechanistic diversity of t...
35 CitationsSource
#1Christopher E. Nelson (Duke University)H-Index: 21
#2Matthew Gemberling (Duke University)H-Index: 12
Last.Charles A. Gersbach (Duke University)H-Index: 39
view all 14 authors...
Duchenne muscular dystrophy (DMD) is a highly prevalent genetic disorder leading to muscle wasting, loss of ambulation, and premature death by the third decade of life. DMD is caused by gene deletions, duplications, or nonsense mutations leading to the loss of dystrophin, an essential musculoskeletal protein. Gene therapy has held tremendous promise for the treatment of monogenic disorders, yet an effective gene replacement therapy has been elusive. Genome editing has been established as a poten...
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#1Christopher E. Nelson (Duke University)H-Index: 21
#2Chady H. Hakim (MU: University of Missouri)H-Index: 16
Last.Charles A. Gersbach (Duke University)H-Index: 39
view all 14 authors...
Duchenne muscular dystrophy (DMD) is a devastating disease affecting about 1 out of 5000 male births and caused by mutations in the dystrophin gene. Genome editing has the potential to restore expression of a modified dystrophin gene from the native locus to modulate disease progression. In this study, adeno-associated virus was used to deliver the clustered regularly interspaced short palindromic repeats (CRISPR)–Cas9 system to the mdx mouse model of DMD to remove the mutated exon 23 from the d...
520 CitationsSource
#2Kexian ZhuH-Index: 6
Last.Feng ZhangH-Index: 115
view all 15 authors...
2 Citations
#1Hiroshi Nishimasu (UTokyo: University of Tokyo)H-Index: 27
#2Le CongH-Index: 16
Last.Osamu Nureki (UTokyo: University of Tokyo)H-Index: 56
view all 10 authors...
Summary The RNA-guided DNA endonuclease Cas9 cleaves double-stranded DNA targets with a protospacer adjacent motif (PAM) and complementarity to the guide RNA. Recently, we harnessed Staphylococcus aureus Cas9 (SaCas9), which is significantly smaller than Streptococcus pyogenes Cas9 (SpCas9), to facilitate efficient in vivo genome editing. Here, we report the crystal structures of SaCas9 in complex with a single guide RNA (sgRNA) and its double-stranded DNA targets, containing the 5′-TTGAAT-3′ PA...
206 CitationsSource
#1F. Ann RanH-Index: 10
#2Le CongH-Index: 16
Last.Feng Zhang (Broad Institute)H-Index: 115
view all 13 authors...
The physical size of the commonly used Cas9 from Streptococcus pyogenes poses challenges for CRISPR-Cas genome editing systems that use the adeno-associated virus as a delivery vehicle; here, smaller Cas9 orthologues are characterized, and Cas9 from Staphylococcus aureus allowed targeting of the cholesterol regulatory gene Pcsk9 in the mouse liver.
1,051 CitationsSource
#1Silvana KonermannH-Index: 13
Last.Feng ZhangH-Index: 115
view all 10 authors...
La presente invention concerne des systemes, des procedes et des compositions destines a modifier l'expression de sequences de genes cibles et de produits de genes afferents. L'invention a egalement trait a des informations structurelles sur la proteine Cas du systeme CRISPR-Cas, a l'utilisation de ces informations dans la production de composants modifies du complexe CRISPR, a des vecteurs et des systemes de vecteurs codant pour un ou plusieurs composants ou composants modifies d'un complexe CR...
#1Feng ZhangH-Index: 115
#2Patrick HsuH-Index: 17
Last.Lukasz SwiechH-Index: 4
view all 5 authors...
Cette invention concerne l'administration, le remaniement et l'optimisation de systemes, de procedes, et de compositions pour la manipulation de sequences et/ou d'activites de sequences cibles. Des vecteurs et des systemes vectoriels, dont certains codent pour un ou plusieurs composants d'un complexe CRISPR, ainsi que des procedes de conception et d'utilisation desdits vecteurs sont en outre decrits. Cette invention concerne egalement des procedes pour diriger la formation d'un complexe CRISPR d...
#1Feng ZhangH-Index: 115
#2Chie-yu LinH-Index: 1
Last.F. Ann RanH-Index: 10
view all 3 authors...
La presente invention concerne, pour l'administration, la modification et l'optimisation de systemes, des methodes et des compositions pour la manipulation de sequences et/ou des activites de sequences cibles. L'invention concerne des vecteurs et des systemes vecteurs, certains codant un ou plusieurs composants d'un complexe CRISPR, ainsi que des methodes pour la conception et l'utilisation de tels vecteurs. L'invention concerne egalement des methodes pour la direction de la formation d'un compl...
#1Mark Lee (MIT: Massachusetts Institute of Technology)H-Index: 21
#2Chun Ye (MIT: Massachusetts Institute of Technology)H-Index: 16
Last.Nir Hacohen (MIT: Massachusetts Institute of Technology)H-Index: 71
view all 24 authors...
Introduction Variation in an individual’s response to environmental factors is likely to influence susceptibility to complex human diseases. The genetic basis of such variation is poorly understood. Here, we identify natural genetic variants that underlie variation in the host innate immune response to infection and analyze the mechanisms by which such variants alter these responses. Identifying the genetic basis of variability in the host response to pathogens. A cohort of 534 individuals donat...
251 CitationsSource
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