Results 121 to 130 of about 1,253,968 (181)

In-depth characterization of stem cell potency and genotoxicity for clinical-scale <i>ex vivo</i> CRISPR-Cas9 gene editing. [PDF]

Mol Ther Adv
Naseem A, Vetharoy W, Whittaker TE, Zinghirino F, Carusillo A, Ghosh R, Gomez-Castaneda E, Ali H, Cipriani C, Montini E, Thrasher AJ, Santilli G, Cesana D, Turchiano G, Cavazza A.   +14 more
europepmc   +1 more source

Gene therapy for genodermatoses at the crossroads of innovation and clinical translation. [PDF]

Front Bioeng Biotechnol
Fabrizi A, Valenti M, Zacchino S, Russo K, Baschetti E, De Rosa L, De Luca M.   +6 more
europepmc   +1 more source

Hearing loss in HDR syndrome

, 2007
openaire   +1 more source

Optimised genome editing for precise DNA insertion and substitution using prime editors in zebrafish. [PDF]

Elife
Ono Y, Peterka M, Love M, Khan A, Bowers F, Bhandari A, Gordon E, Ball JS, Hammond C, Tyler CR, Rees S, Bohlooly-Y M, Maresca M, Scholpp S.   +13 more
europepmc   +1 more source

Precision base-editing of the cryptic 3' acceptor site to correct the RNA splicing defect of β⁶⁵⁴-thalassemia. [PDF]

Mol Biomed
Lu D, Gong X, Guo X, Cai Q, Li W, Chen Y, Li D, Zeng F.   +7 more
europepmc   +1 more source

Engineering the Universal Donor: CRISPR-Mediated Blood Group Antigen Deletion and the Path Toward Truly Universal Red Blood Cells-A Narrative Review. [PDF]

Diagnostics (Basel)
Altayar MA.
europepmc   +1 more source

Host Barriers to In Vivo HSC Gene Therapy with Helper-Dependent Adenovirus Vectors. [PDF]

Viruses
Anderson AK, Lieber A, Li C.
europepmc   +1 more source

Successful Interstitial HDR Brachytherapy for Nasal Skin SCC in Xeroderma Pigmentosum: An Eight-Year Follow-Up Case Report. [PDF]

Clin Case Rep
Saraei P, Derakhshandeh V, Bagheri A.
europepmc   +1 more source