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Engineered compact pan-neuronal promoter from Alphaherpesvirus LAP2 enhances target gene expression in the mouse brain and reduces tropism in the liver
Engineered compact pan-neuronal promoter from Alphaherpesvirus LAP2 enhances target gene expression in the mouse brain and reduces tropism in the liver
Small promoters capable of driving potent neuron-restricted gene expression are required to support successful brain circu...
Looking ahead: ethical and social challenges of somatic gene therapy for sickle cell disease in Africa
Somatic gene therapy will be one of the most exciting practices of genetic medicine in Africa and is primed to offer a ...
Modeling Glutaric Aciduria Type I in human neuroblastoma cells recapitulates neuronal damage that can be rescued by gene replacement
Modeling Glutaric Aciduria Type I in human neuroblastoma cells recapitulates neuronal damage that can be rescued by gene replacement
Glutaric Aciduria type I (GA1) is a rare neurometabolic disorder caused by mutations in the GDCH gene encoding for glutary...
Towards affordable CRISPR genomic therapies: a task force convened by the Innovative Genomics Institute
Towards affordable CRISPR genomic therapies: a task force convened by the Innovative Genomics Institute
To the best of our knowledge, this effort represents the most comprehensive attempt to understand and develop solutions to...
Overexpression of KCNN4 channels in principal neurons produces an anti-seizure effect without reducing their coding ability
Overexpression of KCNN4 channels in principal neurons produces an anti-seizure effect without reducing their coding ability
Gene therapy offers a potential alternative to the surgical treatment of epilepsy, which affects millions of people and is...
Implications of maternal-fetal health on perinatal stem cell banking
Implications of maternal-fetal health on perinatal stem cell banking
Cell based therapies are being assessed for their therapeutic potential across a variety of diseases. Gestational tissues ...
A practical approach for adoption of a hub and spoke model for cell and gene therapies in low- and middle-income countries: framework and case studies
A practical approach for adoption of a hub and spoke model for cell and gene therapies in low- and middle-income countries: framework and case studies
In the rapidly evolving landscape of biotechnologies, cell and gene therapies are being developed and adopted at an unprec...
Cost of gene therapy
Since its earliest days and during the ensuing 5–6 decades, the field of human gene therapy has been char...
Approval and therapeutic value of gene therapies in the US and Europe
Gene therapies are a fast-growing area of innovation and hold promise for the treatment of many diseases currently with un...
Access to affordable medicines: obligations of universities and academic medical centers
The authors thank Katharine Gleason, MPH and Mariah Johnson, MPH for research assistance and administrative support. The a...
Successes and challenges in clinical gene therapy
Despite the ups and downs in the field over three decades, the science of gene therapy has continued to advance and provid...
The estimated annual financial impact of gene therapy in the United States
The estimated annual financial impact of gene therapy in the United States
Gene therapy is a new class of medical treatment that alters part of a patient’s genome through the replacement, del...
Prednisolone and rapamycin reduce the plasma cell gene signature and may improve AAV gene therapy in cynomolgus macaques
Prednisolone and rapamycin reduce the plasma cell gene signature and may improve AAV gene therapy in cynomolgus macaques
Adeno-associated virus (AAV) vector gene therapy is a promising approach to treat rare genetic diseases; however, an ongoi...
p53 dry gene powder enhances anti-cancer effects of chemotherapy against malignant pleural mesothelioma
p53 dry gene powder enhances anti-cancer effects of chemotherapy against malignant pleural mesothelioma
Dry gene powder is a novel non-viral gene-delivery system, which is inhalable with high gene expression. Previously, we sh...
Artificial microRNA suppresses C9ORF72 variants and decreases toxic dipeptide repeat proteins in vivo
Artificial microRNA suppresses C9ORF72 variants and decreases toxic dipeptide repeat proteins in vivo
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease that affects motor neurons, causing progressive m...
Acoustically targeted noninvasive gene therapy in large brain volumes
Acoustically targeted noninvasive gene therapy in large brain volumes
Focused Ultrasound Blood-Brain Barrier Opening (FUS-BBBO) can deliver adeno-associated viral vectors (AAVs) to treat genet...
Long-term effects of a fat-directed FGF21 gene therapy in aged female mice
Long-term effects of a fat-directed FGF21 gene therapy in aged female mice
Fibroblast growth factor 21 (FGF21) has been developed as a potential therapeutic agent for metabolic syndromes. Moreover,...
A pilot study to determine the optimal dose of scAAVIL-1ra in a large animal model of post-traumatic osteoarthritis
A pilot study to determine the optimal dose of scAAVIL-1ra in a large animal model of post-traumatic osteoarthritis
Gene therapy approaches using adeno-associated viral vectors have been successfully tested in the equine post-traumatic os...
Selection of appropriate non-clinical animal models to ensure translatability of novel AAV-gene therapies to the clinic
Selection of appropriate non-clinical animal models to ensure translatability of novel AAV-gene therapies to the clinic
Gene Therapy Medicinal Products consist of a recombinant nucleic acid intended for the modulation or manipulation of a gen...
Safety and biodistribution of XC001 (encoberminogene rezmadenovec) gene therapy in rats: a potential therapy for cardiovascular diseases
Safety and biodistribution of XC001 (encoberminogene rezmadenovec) gene therapy in rats: a potential therapy for cardiovascular diseases
Adenovirus-mediated gene therapy holds promise for the treatment of cardiovascular diseases such as refractory angina. How...
Preclinical safety and biodistribution of CRISPR targeting SIV in non-human primates
Preclinical safety and biodistribution of CRISPR targeting SIV in non-human primates
In this study, we demonstrate the safety and utility of CRISPR-Cas9 gene editing technology for in vivo editing of provira...
Biodistribution of lentiviral transduced adipose-derived stem cells for “ex-vivo” regional gene therapy for bone repair
Biodistribution of lentiviral transduced adipose-derived stem cells for “ex-vivo” regional gene therapy for bone repair
Ex-vivo gene therapy has been shown to be an effective method for treating bone defects in pre-clinical models. As gene th...
Targeted shock-and-kill HIV-1 gene therapy approach combining CRISPR activation, suicide gene tBid and retargeted adenovirus delivery
Targeted shock-and-kill HIV-1 gene therapy approach combining CRISPR activation, suicide gene tBid and retargeted adenovirus delivery
Infections with the human immunodeficiency virus type 1 (HIV-1) are incurable due the long-lasting, latent viral reservoir...
CRISPR/sgRNA-directed synergistic activation mediator (SAM) as a therapeutic tool for Parkinson´s disease
CRISPR/sgRNA-directed synergistic activation mediator (SAM) as a therapeutic tool for Parkinson´s disease
Parkinson`s disease (PD) is the second most prevalent neurodegenerative disease, and different gene therapy strategies hav...
Correction: Site-specific modifications to AAV8 capsid yields enhanced brain transduction in the neonatal MPS IIIB mouse
Authors and AffiliationsDepartment of Medicine, University of Florida College of Medicine, Gainesville, FL, USAJanine A. G...
Sildenafil increases AAV9 transduction after a systemic administration and enhances AAV9-dystrophin therapeutic effect in mdx mice
Sildenafil increases AAV9 transduction after a systemic administration and enhances AAV9-dystrophin therapeutic effect in mdx mice
Adeno-associated virus (AAV) vectors have been successfully used to deliver genes for treating rare diseases. However, the...
Immunobiology of a rationally-designed AAV2 capsid following intravitreal delivery in mice
Immunobiology of a rationally-designed AAV2 capsid following intravitreal delivery in mice
Adeno-associated virus serotype 2 (AAV2) is a viral vector that can be used to deliver therapeutic genes to diseased cells...
Enhanced expression of the human Survival motor neuron 1 gene from a codon-optimised cDNA transgene in vitro and in vivo
Enhanced expression of the human Survival motor neuron 1 gene from a codon-optimised cDNA transgene in vitro and in vivo
Spinal muscular atrophy (SMA) is a neuromuscular disease particularly characterised by degeneration of ventral motor neuro...
Tolerability and tropism of recombinant adeno-associated virus vectors in the African green monkey (Chlorocebus sabaeus) anterior chamber
Tolerability and tropism of recombinant adeno-associated virus vectors in the African green monkey (Chlorocebus sabaeus) anterior chamber
While many studies have investigated the use of recombinant adeno-associated vectors (rAAV) in the posterior chamber for t...
RapaCaspase-9-based suicide gene applied to the safety of IL-1RAP CAR-T cells
RapaCaspase-9-based suicide gene applied to the safety of IL-1RAP CAR-T cells
Even if adoptive cell transfer (ACT) has already shown great clinical efficiency in different types of disease, such as ca...
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