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Items: 1 to 20 of 147


Non-viral delivery systems for CRISPR/Cas9-based genome editing: Challenges and opportunities.

Li L, Hu S, Chen X.

Biomaterials. 2018 Jul;171:207-218. doi: 10.1016/j.biomaterials.2018.04.031. Epub 2018 Apr 18. Review.


Targeted in vivo knock-in of human alpha-1-antitrypsin cDNA using adenoviral delivery of CRISPR/Cas9.

Stephens CJ, Kashentseva E, Everett W, Kaliberova L, Curiel DT.

Gene Ther. 2018 Apr;25(2):139-156. doi: 10.1038/s41434-018-0003-1. Epub 2018 Mar 27.


Induction of apoptosis in imatinib sensitive and resistant chronic myeloid leukemia cells by efficient disruption of bcr-abl oncogene with zinc finger nucleases.

Huang N, Huang Z, Gao M, Luo Z, Zhou F, Liu L, Xiao Q, Wang X, Feng W.

J Exp Clin Cancer Res. 2018 Mar 20;37(1):62. doi: 10.1186/s13046-018-0732-4.


Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression.

Vidal P, Pagliarani S, Colella P, Costa Verdera H, Jauze L, Gjorgjieva M, Puzzo F, Marmier S, Collaud F, Simon Sola M, Charles S, Lucchiari S, van Wittenberghe L, Vignaud A, Gjata B, Richard I, Laforet P, Malfatti E, Mithieux G, Rajas F, Comi GP, Ronzitti G, Mingozzi F.

Mol Ther. 2018 Mar 7;26(3):890-901. doi: 10.1016/j.ymthe.2017.12.019. Epub 2017 Dec 28.


Emerging Issues in AAV-Mediated In Vivo Gene Therapy.

Colella P, Ronzitti G, Mingozzi F.

Mol Ther Methods Clin Dev. 2017 Dec 1;8:87-104. doi: 10.1016/j.omtm.2017.11.007. eCollection 2018 Mar 16. Review.


Hemophilia gene therapy comes of age.

George LA.

Blood Adv. 2017 Dec 8;1(26):2591-2599. doi: 10.1182/bloodadvances.2017009878. eCollection 2017 Dec 12. Review.


In vivo genome editing improves motor function and extends survival in a mouse model of ALS.

Gaj T, Ojala DS, Ekman FK, Byrne LC, Limsirichai P, Schaffer DV.

Sci Adv. 2017 Dec 20;3(12):eaar3952. doi: 10.1126/sciadv.aar3952. eCollection 2017 Dec.


Targeted silencing of SOX2 by an artificial transcription factor showed antitumor effect in lung and esophageal squamous cell carcinoma.

Yokota E, Yamatsuji T, Takaoka M, Haisa M, Takigawa N, Miyake N, Ikeda T, Mori T, Ohno S, Sera T, Fukazawa T, Naomoto Y.

Oncotarget. 2017 Oct 5;8(61):103063-103076. doi: 10.18632/oncotarget.21523. eCollection 2017 Nov 28.


Nuclease-Mediated Gene Therapies for Inherited Metabolic Diseases of the Liver.

Bryson TE, Anglin CM, Bridges PH, Cottle RN.

Yale J Biol Med. 2017 Dec 19;90(4):553-566. eCollection 2017 Dec. Review.


Human genetic variation alters CRISPR-Cas9 on- and off-targeting specificity at therapeutically implicated loci.

Lessard S, Francioli L, Alfoldi J, Tardif JC, Ellinor PT, MacArthur DG, Lettre G, Orkin SH, Canver MC.

Proc Natl Acad Sci U S A. 2017 Dec 26;114(52):E11257-E11266. doi: 10.1073/pnas.1714640114. Epub 2017 Dec 11.


Assembly of CRISPR ribonucleoproteins with biotinylated oligonucleotides via an RNA aptamer for precise gene editing.

Carlson-Stevermer J, Abdeen AA, Kohlenberg L, Goedland M, Molugu K, Lou M, Saha K.

Nat Commun. 2017 Nov 23;8(1):1711. doi: 10.1038/s41467-017-01875-9.


NLRP10 Enhances CD4+ T-Cell-Mediated IFNγ Response via Regulation of Dendritic Cell-Derived IL-12 Release.

Vacca M, Böhme J, Zambetti LP, Khameneh HJ, Paleja BS, Laudisi F, Ho AWS, Neo K, Leong KWK, Marzuki M, Lee B, Poidinger M, Santambrogio L, Tsenova L, Zolezzi F, De Libero G, Singhal A, Mortellaro A.

Front Immunol. 2017 Nov 2;8:1462. doi: 10.3389/fimmu.2017.01462. eCollection 2017.


CRISPR Genome Engineering for Human Pluripotent Stem Cell Research.

Chaterji S, Ahn EH, Kim DH.

Theranostics. 2017 Oct 7;7(18):4445-4469. doi: 10.7150/thno.18456. eCollection 2017. Review.


Disruption of diphthamide synthesis genes and resulting toxin resistance as a robust technology for quantifying and optimizing CRISPR/Cas9-mediated gene editing.

Killian T, Dickopf S, Haas AK, Kirstenpfad C, Mayer K, Brinkmann U.

Sci Rep. 2017 Nov 13;7(1):15480. doi: 10.1038/s41598-017-15206-x.


Combining Engineered Nucleases with Adeno-associated Viral Vectors for Therapeutic Gene Editing.

Epstein BE, Schaffer DV.

Adv Exp Med Biol. 2017;1016:29-42. doi: 10.1007/978-3-319-63904-8_2. Review.


CRISPR-Cas9 Genome Editing for Treatment of Atherogenic Dyslipidemia.

Chadwick AC, Musunuru K.

Arterioscler Thromb Vasc Biol. 2018 Jan;38(1):12-18. doi: 10.1161/ATVBAHA.117.309326. Epub 2017 Aug 24. Review.


Engineering and Application of Zinc Finger Proteins and TALEs for Biomedical Research.

Kim MS, Kini AG.

Mol Cells. 2017 Aug;40(8):533-541. doi: 10.14348/molcells.2017.0139. Epub 2017 Aug 23. Review.


CRISPR/Cas9-mediated genome editing via postnatal administration of AAV vector cures haemophilia B mice.

Ohmori T, Nagao Y, Mizukami H, Sakata A, Muramatsu SI, Ozawa K, Tominaga SI, Hanazono Y, Nishimura S, Nureki O, Sakata Y.

Sci Rep. 2017 Jun 23;7(1):4159. doi: 10.1038/s41598-017-04625-5.


Gene Therapy for Hemophilia.

Nienhuis AW, Nathwani AC, Davidoff AM.

Mol Ther. 2017 May 3;25(5):1163-1167. doi: 10.1016/j.ymthe.2017.03.033. Epub 2017 Apr 11. Review.


Genome editing for inborn errors of metabolism: advancing towards the clinic.

Schneller JL, Lee CM, Bao G, Venditti CP.

BMC Med. 2017 Feb 27;15(1):43. doi: 10.1186/s12916-017-0798-4. Review.

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