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Proceedings of the National Academy of Sciences of the United States of America, ISSN 0027-8424, 12/2017, Volume 114, Issue 51, pp. 13567 - 13572
Journal Article
Molecular Therapy - Nucleic Acids, ISSN 2162-2531, 09/2019, Volume 17, pp. 198 - 209
Given that the cDNA of is too large to be packaged into adeno-associated virus (AAV) capsids, gene transfer of some versions of B-domain-deleted (BDD- ) for... 
hemophilia A | iPSCs | gene therapy | ssODN | CRISPR/Cas9 | in-frame deletion
Journal Article
Molecular Therapy - Nucleic Acids, ISSN 2162-2531, 2012, Volume 1, Issue 12, pp. e59 - e59
Gene editing is a process by which single base mutations can be corrected, in the context of the chromosome, using single-stranded oligodeoxynucleotides... 
ssODN | electrospun | gene editing | nanofiber | Nanofiber | Electrospun | Gene editing | MEDICINE, RESEARCH & EXPERIMENTAL | Methods - Original
Journal Article
Molecular Therapy - Nucleic Acids, ISSN 2162-2531, 09/2019, Volume 17, pp. 277 - 288
Gene editing following designer nuclease cleavage in the presence of a DNA donor template can revert mutations in disease-causing genes. For optimal benefit,... 
stem cell cures | NBSGW41 mice | gene editing | hemoglobin disorders | in vivo engraftment | sickle cell disease | ssODN | NHEJ versus HDR | rAAV6 | Crispr/Cas9 | homology-directed repair | hematopoietic stem cells | CD34
Journal Article
Animal Biotechnology, ISSN 1049-5398, 07/2017, Volume 28, Issue 3, pp. 198 - 210
In the past few years, new technologies have arisen that enable higher efficiency of gene editing. With the increase ease of using gene editing technologies,... 
NHEJ, non-homologous end joining | RVDs, repeat variable diresidues | ssODN, single strand oligo nucleotide | PAM, protospacer adjacent motif | iPSC, induced pluripotent stem cells | ICM, inner cell mass | EG, embryonic germ | SMGT, sperm mediated gene transfer | HDR, homology directed repair | TALEN | CRISPRs, clustered regulatory interspaced short palindromic repeats | OBCT, oocyte bisection technique | PGCS, primordial germ cells | SV40, simian virus 40 | CRISPR | SMGT | ZFN | gene transfer | BLG, β-lactoglobulin | NT, nuclear transfers | ZFN, Zinc-finger nucleases | TALENs, transcription activator-like effector nucleases | ICSI, intracytoplasmic sperm injection | ESC, Embryonic stem cell | gene editing | ES, Embryonic stem | PCR, polymerase chain reaction | nuclear transfer | FULL-TERM DEVELOPMENT | PGCS | KNOCKOUT PIGS | Zinc-finger nucleases | TALENs | protospacer adjacent motif | transcription activator-like effector nucleases | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | primordial germ cells | single strand oligo nucleotide | OBCT | embryonic germ | iPSC | MOUSE EMBRYOS | MEDIATED GENE-TRANSFER | PRIMORDIAL GERM-CELLS | ssODN | SV40 | non-homologous end joining | induced pluripotent stem cells | simian virus 40 | BLG | PCR | homology directed repair | lactoglobulin | sperm mediated gene transfer | inner cell mass | intracytoplasmic sperm injection | repeat variable diresidues | ICSI | ESC | BETA-GLOBIN GENE | RVDs | HDR | EMBRYONIC STEM-CELLS | polymerase chain reaction | CRISPRs | AGRICULTURE, DAIRY & ANIMAL SCIENCE | Embryonic stem cell | NUCLEAR TRANSPLANTATION | EFFICIENT GENERATION | Embryonic stem | oocyte bisection technique | ICM | FOREIGN DNA | nuclear transfers | clustered regulatory interspaced short palindromic repeats | NHEJ | PAM | Gene Editing - methods | Animals | Livestock - genetics | Animals, Genetically Modified - genetics | CRISPR-Cas Systems | Genome - genetics | Accessibility | Transcription | Gene transfer | Cloning | Editing | Animal diseases | Genomes | Domestic animals | Zinc | Biomedical materials | Microinjection | Transgenic animals | Nuclear transfer | Mice | Livestock | New technology | Genetic modification | Methods | Deoxyribonucleic acid--DNA | Index Medicus
Journal Article
Methods in Molecular Biology, ISSN 1064-3745, 2017, Volume 1630, pp. 91 - 100
Journal Article
Insect Biochemistry and Molecular Biology, ISSN 0965-1748, 10/2018, Volume 101, pp. 85 - 93
Journal Article
G3 (Bethesda, Md.), ISSN 2160-1836, 01/2019, Volume 9, Issue 1, pp. 281 - 286
Journal Article
Frontiers in physiology, ISSN 1664-042X, 2014, Volume 5, p. 148
The progressive loss of muscle mass characteristic of many muscular dystrophies impairs the efficacy of most of the gene and molecular therapies currently... 
Journal Article
Molecular Therapy - Nucleic Acids, ISSN 2162-2531, 03/2017, Volume 6, Issue C, pp. 57 - 67
Monogenic disorders (MGDs), which are caused by single gene mutations, have a serious effect on human health. Among these, β-thalassemia (β-thal) represents... 
β thalassemia | induced pluripotent stem cells | CRISPR/Cas9 | ssODNs | TARGET | MEDICINE, RESEARCH & EXPERIMENTAL | CELLS | CRISPR/CAS9 | THERAPY | GENE CORRECTION | TRANSPLANTATION | CRISPR | Cas9 | Original
Journal Article
Biochemistry and Biophysics Reports, ISSN 2405-5808, 03/2019, Volume 17, pp. 81 - 86
Alport syndrome (AS) is an inherited disorder characterized by glomerular basement membrane (GBM) abnormality and development of chronic kidney disease at an... 
ESRD | Col4a5 | Model mice | CKD | Alport syndrome | XLAS
Journal Article