@article {46331,
	title = {Francisella novicida Cas9 interrogates genomic DNA with very high specificity and can be used for mammalian genome editing},
	journal = {Proceedings of the National Academy of Sciences of the United States of America},
	volume = {116},
	year = {2019},
	month = {OCT},
	pages = {20959-20968},
	type = {Article},
	abstract = {<p>Genome editing using the CRISPR/Cas9 system has been used to make precise heritable changes in the DNA of organisms. Although the widely used Streptococcus pyogenes Cas9 (SpCas9) and its engineered variants have been efficiently harnessed for numerous gene-editing applications across different platforms, concerns remain regarding their putative off-targeting at multiple loci across the genome. Here we report that Francisella novicida Cas9 (FnCas9) shows a very high specificity of binding to its intended targets and negligible binding to off-target loci. The specificity is determined by its minimal binding affinity with DNA when mismatches to the target single-guide RNA (sgRNA) are present in the sgRNA:DNA heteroduplex. FnCas9 produces staggered cleavage, higher homology-directed repair rates, and very low nonspecific genome editing compared to SpCas9. We demonstrate FnCas9-mediated correction of the sickle cell mutation in patient-derived induced pluripotent stem cells and propose that it can be used for precise therapeutic genome editing for a wide variety of genetic disorders.</p>
},
	keywords = {CRISPR Cas9, gene therapy, Genome editing, iPSCs, sickle cell anemia},
	issn = {0027-8424},
	doi = {10.1073/pnas.1818461116},
	author = {Acharya, Sundaram and Mishra, Arpit and Paul, Deepanjan and Ansari, Asgar Hussain and Azhar, Mohd and Kumar, Manoj and Rauthan, Riya and Sharma, Namrata and Aich, Meghali and Sinha, Dipanjali and Sharma, Saumya and Jain, Shivani and Ray, Arjun and Jain, Suman and Ramalingam, Sivaprakash and Maiti, Souvik and Chakraborty, Debojyoti}
}
@article {48068,
	title = {Postmodification of voxelotor (GBT 440) via [Rh]-catalyzed cross dehydrogenative coupling with olefins br},
	journal = {Bioorganic \& Medicinal Chemistry Letters},
	volume = {77},
	year = {2022},
	month = {DEC},
	pages = {129022},
	type = {Article},
	abstract = {<p>
	The directed Rh(III)-catalyzed cross dehydrogenative coupling of the pyrazole unit of the GBT-440 scaffold has been explored with simple as well as conjugated olefins to synthesize post-functionalized GBT-440 analogues. The screening of these synthesized compounds for improving the oxygen binding efficiency of the hemoglobin isolated from the sickled red blood cells revealed that some of these compounds are as good as GBT-440.</p>
},
	keywords = {CDC Coupling, GBT-440 (Voxelotor), Rh(III) catalysis, sickle cell anemia},
	issn = {0960-894X},
	doi = {10.1016/j.bmcl.2022.129022},
	author = {Kalshetti, Rupali G. and Rajput, Raveena and Motiwala, Zenia and Reddy, D. Srinivasa and Kulkarni, Kiran A. and V. Ramana, Chepuri}
}
