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                             66 results found
no title author magazine year volume issue page(s) type
1 AAV2-antiVEGFscFv gene therapy for retinal neovascularization Han, Ni
31 C p.
 article
2 AAV-BDNF gene therapy ameliorates a hypothalamic neuroinflammatory signature in the Magel2-null model of Prader-Willi syndrome Queen, Nicholas J.
31 C p.
 article
3 A C1qTNF3 collagen domain fusion chaperones diverse secreted proteins and anti-Aβ scFvs: Applications for gene therapies Moore, Brenda D.
31 C p.
 article
4 Adeno-associated viral capsid stability on anion exchange chromatography column and its impact on empty and full capsid separation Khanal, Ohnmar
31 C p.
 article
5 Adeno-associated virus serotype 9 antibody seroprevalence for patients in the United States with spinal muscular atrophy Day, John W.
31 C p.
 article
6 Analysis of vector genome integrations in multicentric lymphoma after AAV gene therapy in a severe hemophilia A dog Van Gorder, Lucas
31 C p.
 article
7 A novel FOXP3 knockout-humanized mouse model for pre-clinical safety and efficacy evaluation of Treg-like cell products Sato, Yohei
31 C p.
 article
8 Anti-tau intrabodies: From anti-tau immunoglobulins to the development of functional scFv intrabodies Rodrigues Martins, Dina
31 C p.
 article
9 Archival skin biopsy specimens as a tool for miRNA-based diagnosis: Technical and post-analytical considerations Andelic, Mirna
31 C p.
 article
10 A sensitive AAV transduction inhibition assay assists evaluation of critical factors for detection and concordance of pre-existing antibodies Pan, Yonghua
31 C p.
 article
11 A T cell-targeted multi-antigen vaccine generates robust cellular and humoral immunity against SARS-CoV-2 infection Boulton, Stephen
31 C p.
 article
12 Awake intracerebroventricular delivery and safety assessment of oligonucleotides in a large animal model Benatti, Hector Ribeiro
31 C p.
 article
13 Brain-targeted ex vivo lentiviral gene therapy: Implications for MPS and beyond Bradbury, Allison M.
31 C p.
 article
14 Breaching the blood-brain barrier: AAV triggers dose-dependent toxicity in the brain Stone, Daniel
31 C p.
 article
15 Bringing base editing to the clinic: The next generation of genome editors Flugel, Christian L.
31 C p.
 article
16 Case report of selumetinib as a novel therapy in a neurofibromatosis type 2-associated ependymoma Blackwood, Nigel
31 C p.
 article
17 Characterization and AAV-mediated CRB gene augmentation in human-derived CRB1 KO and CRB1 KO CRB2 +/− retinal organoids Boon, Nanda
31 C p.
 article
18 Clinical holds for cell and gene therapy trials: Risks, impact, and lessons learned Wills, Carolyn A.
31 C p.
 article
19 CRISPR-Cas9-based non-viral gene editing therapy for topical treatment of recessive dystrophic epidermolysis bullosa Wang, Xianqing
31 C p.
 article
20 CRISPR-Cas9-mediated somatic correction of a one-base deletion in the Ugt1a gene ameliorates hyperbilirubinemia in Crigler-Najjar syndrome mice Bortolussi, Giulia
31 C p.
 article
21 Cytokine and reactivity profiles in SLE patients following anti-CD19 CART therapy Nunez, Daniel
31 C p.
 article
22 Development of a gene edited next-generation hematopoietic cell transplant to enable acute myeloid leukemia treatment by solving off-tumor toxicity Lydeard, John R.
31 C p.
 article
23 Development of a gene edited next-generation hematopoietic cell transplant to enable acute myeloid leukemia treatment by solving off-tumor toxicity Lydeard, John R.
31 C p.
 article
24 Development of an automated manufacturing process for large-scale production of autologous T cell therapies Francis, Natalie
31 C p.
 article
25 Development of capsid- and genome-modified optimized AAVrh74 vectors for muscle gene therapy Shoti, Jakob
31 C p.
 article
26 Drug product attributes predict clinical efficacy in betibeglogene autotemcel gene therapy for β-thalassemia Whitney, Dustin
31 C p.
 article
27 Efficient long-term multilineage engraftment of CD33-edited hematopoietic stem/progenitor cells in nonhuman primates Petty, Nicholas E.
31 C p.
 article
28 Engineering a highly durable adeno-associated virus receptor for analytical applications Yoshida, Kouhei
31 C p.
 article
29 Enhancement of recombinant adeno-associated virus activity by improved stoichiometry and homogeneity of capsid protein assembly Onishi, Takayuki
31 C p.
 article
30 Fast HPLC-based affinity method to determine capsid titer and full/empty ratio of adeno-associated viral vectors Heckel, Jakob
31 C p.
 article
31 Generation of hepatitis C virus–resistant liver cells by genome editing–mediated stable expression of RNA aptamer Kim, Tae Hyeong
31 C p.
 article
32 Genome length determination in adeno-associated virus vectors with mass photometry Hiemenz, Cornelia
31 C p.
 article
33 Hepatocyte-targeted siTAZ therapy lowers liver fibrosis in NASH diet-fed chimeric mice with hepatocyte-humanized livers Wang, Xiaobo
31 C p.
 article
34 High-titer AAV disrupts cerebrovascular integrity and induces lymphocyte infiltration in adult mouse brain Guo, Yaowei
31 C p.
 article
35 Identification of a small molecule for enhancing lentiviral transduction of T cells Malach, Paulina
31 C p.
 article
36 Immortalized human myoblast cell lines for the delivery of therapeutic proteins using encapsulated cell technology Lathuiliere, Aurelien
31 C p.
 article
37 iMSC: One leap closer to clinical applications Hoang, Duc M.
31 C p.
 article
38 Inducible caspase 9-mediated suicide gene therapy using AAV6 vectors in a murine model of breast cancer Pathak, Subhajit
31 C p.
 article
39 Inhibition of DNA-dependent protein kinase catalytic subunit boosts rAAV transduction of polarized human airway epithelium Ning, Kang
31 C p.
 article
40 Iron oxide-coupled CRISPR-nCas9-based genome editing assessment in mucopolysaccharidosis IVA mice Leal, Andrés Felipe
31 C p.
 article
41 Large-scale manufacturing of base-edited chimeric antigen receptor T cells Woodruff, Rosie
31 C p.
 article
42 Lethality rescue and long-term amelioration of a citrullinemia type I mouse model by neonatal gene-targeting combined to SaCRISPR-Cas9 Lisjak, Michela
31 C p.
 article
43 Lipid nanoparticles outperform electroporation in mRNA-based CAR T cell engineering Kitte, Reni
31 C p.
 article
44 Long-term dystrophin restoration supports development of splice correction therapy for DMD patients with exon 2 duplications Roberts, Thomas C.
31 C p.
 article
45 Mini-TCRs: Truncated T cell receptors to generate T cells from induced pluripotent stem cells Takayanagi, Shin-ichiro
31 C p.
 article
46 Mitochondrial transplantation reduces lower limb ischemia-reperfusion injury by increasing skeletal muscle energy and adipocyte browning Zeng, Jiaqi
31 C p.
 article
47 Molecular and functional correction of a deep intronic splicing mutation in CFTR by CRISPR-Cas9 gene editing Walker, Amy J.
31 C p.
 article
48 Multiparametric domain insertional profiling of adeno-associated virus VP1 Hoffmann, Mareike D.
31 C p.
 article
49 New dawn of cellular therapies in autoimmune diseases Wagner, Dimitrios Laurin
31 C p.
 article
50 Non-canonical amino acid incorporation into AAV5 capsid enhances lung transduction in mice Chang, Hao
31 C p.
 article
51 Optogenetic targeting of AII amacrine cells restores retinal computations performed by the inner retina Khabou, Hanen
31 C p.
 article
52 Oxidative stress induced by sustained supraphysiological intrastriatal GDNF delivery is prevented by dose regulation Azevedo, Marcelo Duarte
31 C p.
 article
53 PCR-based analytics of gene therapies using adeno-associated virus vectors: Considerations for cGMP method development Blay, Emmanuel
31 C p.
 article
54 Persistence of exon 2 skipping and dystrophin expression at 18 months after U7snRNA-mediated therapy in the Dup2 mouse model Gushchina, Liubov V.
31 C p.
 article
55 Preclinical evaluation of the efficacy and safety of AAV1-hOTOF in mice and nonhuman primates Zhang, Longlong
31 C p.
 article
56 Predicted deleterious variants in the human genome relevant to gene therapy with adeno-associated virus vectors Rostami, Mahboubeh R.
31 C p.
 article
57 Production of therapeutic levels of human FIX-R338L by engineered B cells using GMP-compatible medium David, Marion
31 C p.
 article
58 Prostaglandin E2 as transduction enhancer affects competitive engraftment of human hematopoietic stem and progenitor cells Poletti, Valentina
31 C p.
 article
59 Rationale for using centralized transduction inhibition assays in three phase 3 rAAV gene therapy clinical trials Schulz, Martin
31 C p.
 article
60 Rescuing the cytolytic function of APDS1 patient T cells via TALEN-mediated PIK3CD gene correction Poggi, Lucie
31 C p.
 article
61 Sustained long-term disease correction in a murine model of MPSII following stem cell gene therapy Ellison, Stuart
31 C p.
 article
62 Tagged IDS causes efficient and engraftment-independent prevention of brain pathology during lentiviral gene therapy for Mucopolysaccharidosis type II Catalano, Fabio
31 C p.
 article
63 Thank you to our 2023 reviewers
31 C p.
 article
64 The hunt for novel AAV capsids with improved cardiac tropism Miguel-dos-Santos, Rodrigo
31 C p.
 article
65 Universal ddPCR-based assay for the determination of lentivirus infectious titer and lenti-modified cell vector copy number Kandell, Jennifer
31 C p.
 article
66 Unveiling the secrets of adeno-associated virus: novel high-throughput approaches for the quantification of multiple serotypes Meierrieks, Frederik
31 C p.
 article
                             66 results found
 
 Koninklijke Bibliotheek - National Library of the Netherlands