title: In Vivo Delivery of CRISPR/Cas9 for Therapeutic Gene Editing: Progress and Challenges.
abstract: The successful use of clustered regularly interspaced short palindromic repeat (CRISPR)/Cas9-based gene editing for therapeutics requires efficient in vivo delivery of the CRISPR components. There are, however, major challenges on the delivery front. In this Topical Review, we will highlight recent developments in CRISPR delivery, and we will present hurdles that still need to be overcome to achieve effective in vivo editing.
authors.affiliation: Department of Chemistry, University of Massachusetts , 710 North Pleasant Street, Amherst, Massachusetts 01003, United States.
authors.forename: Rubul
authors.identifier: http://orcid.org/0000-0001-6125-5873
authors.initials: R
authors.lastname: Mout
authors.affiliation: Department of Chemistry, University of Massachusetts , 710 North Pleasant Street, Amherst, Massachusetts 01003, United States.
authors.forename: Moumita
authors.initials: M
authors.lastname: Ray
authors.affiliation: Department of Chemistry, University of Massachusetts , 710 North Pleasant Street, Amherst, Massachusetts 01003, United States.
authors.forename: Yi-Wei
authors.initials: YW
authors.lastname: Lee
authors.affiliation: Department of Chemistry, University of Massachusetts , 710 North Pleasant Street, Amherst, Massachusetts 01003, United States.
authors.forename: Federica
authors.initials: F
authors.lastname: Scaletti
authors.affiliation: Department of Chemistry, University of Massachusetts , 710 North Pleasant Street, Amherst, Massachusetts 01003, United States.
authors.forename: Vincent M
authors.identifier: http://orcid.org/0000-0002-5184-5439
authors.initials: VM
authors.lastname: Rotello
mesh_terms: D000818:Animals; D064113:CRISPR-Cas Systems; D064112:Clustered Regularly Interspaced Short Palindromic Repeats; D000072669:Gene Editing / Q000379:methods*; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods; D005822:Genetic Vectors / Q000008:administration & dosage / Q000235:genetics; D006801:Humans; D016254:Mutagenesis, Insertional / Q000379:methods; D014780:Viruses / Q000235:genetics