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Perspective
| Open AccessPrecision ophthalmology: a call for Africa not to be left in the dark
- Lisa Roberts
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Brief Communication
| Open Access
A shedding analysis after AAV8 CNS injection revealed fragmented viral DNA without evidence of functional AAV particles in mice
- Felix Krause
- , Katja Schmidtke
- & Max O. Rybarski
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Article
| Open Access
Analytical characterization of full, intermediate, and empty AAV capsids
- Aisleen McColl-Carboni
- , Serena Dollive
- & James B. McGivney IV
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Article |
Development of a stable Sf9 insect cell line to produce VSV-G pseudotyped baculoviruses
- María del Pilar Plastine
- , Sabrina Amalfi
- & Victoria Alfonso
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Article
| Open Access
Ghrelin mediated cardioprotection using in vitro models of oxidative stress
- Cindy Y. Kok
- , George Ghossein
- & Eddy Kizana
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Article |
Distributional comparison of different AAV vectors after unilateral cochlear administration
- Shuang Han
- , Zhijiao Xu
- & Yilai Shu
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Brief Communication |
Modeling Glutaric Aciduria Type I in human neuroblastoma cells recapitulates neuronal damage that can be rescued by gene replacement
- A. Mateu-Bosch
- , E. Segur-Bailach
- & C. Fillat
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Perspective
| Open Access
Towards affordable CRISPR genomic therapies: a task force convened by the Innovative Genomics Institute
- Lea Witkowsky
- , Matthew Norstad
- & Melinda Kliegman
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Article
| Open Access
The estimated annual financial impact of gene therapy in the United States
- Chi Heem Wong
- , Dexin Li
- & Rena M. Conti
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Review Article
| Open Access
Implications of maternal-fetal health on perinatal stem cell banking
- Dandan Zhu
- , Mehri Barabadi
- & Rebecca Lim
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Article |
Long-term effects of a fat-directed FGF21 gene therapy in aged female mice
- Jacqueline M. Anderson
- , W. David Arnold
- & Lei Cao
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Review Article |
Selection of appropriate non-clinical animal models to ensure translatability of novel AAV-gene therapies to the clinic
- Mark Singh
- , Andrew Brooks
- & Keith McLuckie
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Article
| Open Access
Targeted shock-and-kill HIV-1 gene therapy approach combining CRISPR activation, suicide gene tBid and retargeted adenovirus delivery
- Sarah Klinnert
- , Corinne D. Schenkel
- & Karin J. Metzner
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Article |
Sildenafil increases AAV9 transduction after a systemic administration and enhances AAV9-dystrophin therapeutic effect in mdx mice
- Kaiyi Zhou
- , Meng Yuan
- & Xia Wu
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Article
| Open Access
Immunobiology of a rationally-designed AAV2 capsid following intravitreal delivery in mice
- Michael Whitehead
- , Andrew Sage
- & Keith R. Martin
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Article |
Enhanced expression of the human Survival motor neuron 1 gene from a codon-optimised cDNA transgene in vitro and in vivo
- Neda A. M. Nafchi
- , Ellie M. Chilcott
- & Rafael J. Yáñez-Muñoz
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Article
| Open Access
Repeat or single-dose lentiviral vector administration to mouse lungs? It’s all about the timing
- Martin Donnelley
- , Patricia Cmielewski
- & Nigel Farrow
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Article
| Open Access
rAAV-PHP.B escapes the mouse eye and causes lethality whereas rAAV9 can transduce aniridic corneal limbal stem cells without lethality
- Seyedeh Zeinab Mirjalili Mohanna
- , Andrea J. Korecki
- & Elizabeth M. Simpson
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Article
| Open Access
Large-scale purification of functional AAV particles packaging the full genome using short-term ultracentrifugation with a zonal rotor
- Mikako Wada
- , Naoya Uchida
- & Takashi Okada
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Article |
Gene augmentation for autosomal dominant retinitis pigmentosa using rhodopsin genomic loci nanoparticles in the P23H+/− knock-in murine model
- Simna SP
- , Rajendra N. Mitra
- & Zongchao Han
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Article
| Open Access
Terminal hairpins improve protein expression in IRES-initiated mRNA in the absence of a cap and polyadenylated tail
- Victor Solodushko
- & Brian Fouty
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Article |
Reversible stabilization of DNA/PEI complexes by reducible click-linkage between DNA and polymer. A new polyplex concept for lowering polymer quantity
- Delphine Maze
- , Chantal Pichon
- & Patrick Midoux
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Article |
Antibody-based protection against respiratory syncytial virus in mice and their offspring through vectored immunoprophylaxis
- Amira D. Rghei
- , Jacob G. E. Yates
- & Sarah K. Wootton
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Review Article |
Empowering gene delivery with protein engineering platforms
- Monika Kizerwetter
- , Kevin Pietz
- & Jamie B. Spangler
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Brief Communication |
Potential of an anti-bevacizumab idiotype scFv DNA-based immunization to elicit VEGF-binding antibody response
- Tábata Almeida Silva
- , Rodrigo Barbosa Aguiar
- & Jane Zveiter Moraes
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Article |
In vivo genome editing using novel AAV-PHP variants rescues motor function deficits and extends survival in a SOD1-ALS mouse model
- Yi A. Chen
- , Mark W. Kankel
- & Shih-Ching Lo
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Article |
A new protocol for whole-brain biodistribution analysis of AAVs by tissue clearing, light-sheet microscopy and semi-automated spatial quantification
- Miguel M. Lopes
- , Jacques Paysan
- & Rui Jorge Nobre
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Article |
IgG-cleavage protein allows therapeutic AAV gene delivery in passively immunized MPS IIIA mice
- Tierra A. Bobo
- , Preston N. Samowitz
- & Haiyan Fu
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Article |
Design, construction and in vivo functional assessment of a hinge truncated sFLT01
- Fahimeh Zakeri
- , Hamid Latifi-Navid
- & Ali Hafezi-Moghadam
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Article |
Changing trends in the development of AAV-based gene therapies: a meta-analysis of past and present therapies
- Tamara Burdett
- & Samir Nuseibeh
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Article
| Open Access
Multiplex viral tropism assay in complex cell populations with single-cell resolution
- Choong Tat Keng
- , Ke Guo
- & Wei Leong Chew
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Article
| Open Access
Preclinical assessment of antigen-specific chimeric antigen receptor regulatory T cells for use in solid organ transplantation
- Emma Proics
- , Marion David
- & Céline Dumont
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Article |
A novel preclinical model of mucopolysaccharidosis type II for developing human hematopoietic stem cell gene therapy
- Yohta Shimada
- , Natsumi Ishii
- & Hiroshi Kobayashi
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Article |
Selective attachment of a microtubule interacting peptide to plasmid DNA via a triplex forming oligonucleotide for transfection improvement
- Caroline Girardin
- , Delphine Maze
- & Patrick Midoux
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Article |
Intracerebral lentiviral ABCD1 gene therapy in an early disease onset ALD mouse model
- Jie Gong
- , Yunyun Liu
- & Lung-Ji Chang
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Article
| Open Access
Clinical enrollment assay to detect preexisting neutralizing antibodies to AAV6 with demonstrated transgene expression in gene therapy trials
- Liching Cao
- , Annemarie Ledeboer
- & Kathleen Meyer
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Article
| Open Access
Enzymatically amplified linear dbDNATM as a rapid and scalable solution to industrial lentiviral vector manufacturing
- Maria Barreira
- , Claire Kerridge
- & Sarah Smith-Moore
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Article |
Efficient CRISPR/Cas9 nickase-mediated genome editing in an in vitro model of mucopolysaccharidosis IVA
- Andrés Felipe Leal
- & Carlos Javier Alméciga-Díaz
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Article |
Epigenetic repression of gonadotropin gene expression via a GnRH-mediated DNA delivery system
- Lilach Pnueli
- & Philippa Melamed
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Article
| Open Access
Lentiviral standards to determine the sensitivity of assays that quantify lentiviral vector copy numbers and genomic insertion sites in cells
- Guillaume Corre
- , Ababacar Seye
- & Anne Galy
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Brief Communication
| Open Access
Lack of germline transmission in male mice following a single intravenous administration of AAV5-hFVIII-SQ gene therapy
- Carlos Fonck
- , Cheng Su
- & Charles A. O’Neill
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Article
| Open Access
AAV2-VEGF-B gene therapy failed to induce angiogenesis in ischemic porcine myocardium due to inflammatory responses
- Henna Korpela
- , Jaakko Lampela
- & Seppo Ylä-Herttuala
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Article |
Evaluation of the dystrophin carboxy-terminal domain for micro-dystrophin gene therapy in cardiac and skeletal muscles in the DMDmdx rat model
- Audrey Bourdon
- , Virginie François
- & Caroline Le Guiner
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Article
| Open Access
First use of gene therapy to treat growth hormone resistant dwarfism in a mouse model
- Kian Chuan Sia
- , Shu Uin Gan
- & Kok Onn Lee
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Article |
Two high-fidelity variants: efSaCas9 and SaCas9-HF, which one is better?
- Jineng Lv
- , Haitao Xi
- & Zongming Song
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Article |
A PCR-amplified transgene fragment flanked by a single copy of a truncated inverted terminal repeat for recombinant adeno-associated virus production prevents unnecessary plasmid DNA packaging
- Kumi Adachi
- , Taro Tomono
- & Takashi Okada
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Article |
Rational engineering of a functional CpG-free ITR for AAV gene therapy
- Xiufang Pan
- , Yongping Yue
- & Dongsheng Duan
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Article
| Open Access
Long-term safety and efficacy of intramyocardial adenovirus-mediated VEGF-DΔNΔC gene therapy eight-year follow-up of phase I KAT301 study
- Aleksi J. Leikas
- , Iiro Hassinen
- & Juha E. K. Hartikainen
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Article |
CRISPR-SCReT (CRISPR-Stop Codon Read Through) method to control Cas9 expression for gene editing
- Pouiré Yaméogo
- , Benjamin L. Duchêne
- & Jacques P. Tremblay
Adeno-associated virus genome quantification with amplification-free CRISPR-Cas12aBrowse broader subjects
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