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Towards ethical drug pricing: the European Orphan Genomic Therapies Fund
An increasing number of novel genomic therapies are expected to become available for patients with rare or ultra-rare dise...
Long-term benefits of hematopoietic stem cell-based macrophage/microglia delivery of GDNF to the CNS in a mouse model of Parkinson’s disease
Long-term benefits of hematopoietic stem cell-based macrophage/microglia delivery of GDNF to the CNS in a mouse model of Parkinson’s disease
Glial cell line-derived neurotrophic factor (GDNF) protects dopaminergic neurons in various models of Parkinson’s di...
A multinational survey of potential participant perspectives on ocular gene therapy
A multinational survey of potential participant perspectives on ocular gene therapy
Amidst rapid advancements in ocular gene therapy, understanding patient perspectives is crucial for shaping future treatme...
Adeno-associated virus genome quantification with amplification-free CRISPR-Cas12a
Adeno-associated virus genome quantification with amplification-free CRISPR-Cas12a
Efficient manufacturing of recombinant Adeno-Associated Viral (rAAV) vectors to meet rising clinical demand remains a majo...
Precision ophthalmology: a call for Africa not to be left in the dark
Russell S, Bennett J, Wellman JA, Chung DC, Yu ZF, Tillman A, et al. Efficacy and safety of voretigene neparvovec (AAV2-hR...
Advancing rare disease treatment: EMA’s decade-long insights into engineered adoptive cell therapy for rare cancers and orphan designation
Advancing rare disease treatment: EMA’s decade-long insights into engineered adoptive cell therapy for rare cancers and orphan designation
Adoptive cell therapy (ACT), particularly chimeric antigen receptor (CAR)-T cell therapy, has emerged as a promising appro...
A shedding analysis after AAV8 CNS injection revealed fragmented viral DNA without evidence of functional AAV particles in mice
A shedding analysis after AAV8 CNS injection revealed fragmented viral DNA without evidence of functional AAV particles in mice
Adeno-associated viruses (AAV) are commonly used in the scientific field due to their diverse application range. However, ...
Are genetically modified protozoa eligible for ATMP status? Concerning the legal categorization of an oncolytic protozoan drug candidate
Are genetically modified protozoa eligible for ATMP status? Concerning the legal categorization of an oncolytic protozoan drug candidate
Neospora caninum is an obligate intracellular protozoan that affects several animal species. It is not pathogenic for huma...
Analytical characterization of full, intermediate, and empty AAV capsids
Analytical characterization of full, intermediate, and empty AAV capsids
Manufacturing of recombinant adeno-associated virus (AAV) vectors produces three types of capsids: full, intermediate, and...
Multicenter assessment and longitudinal study of the prevalence of antibodies and related adaptive immune responses to AAV in adult males with hemophilia
Multicenter assessment and longitudinal study of the prevalence of antibodies and related adaptive immune responses to AAV in adult males with hemophilia
Adeno-associated virus (AAV) based gene therapy has demonstrated effective disease control in hemophilia. However, pre-exi...
Gene therapy corrects the neurological deficits of mice with sialidosis
Gene therapy corrects the neurological deficits of mice with sialidosis
Patients with sialidosis (mucolipidosis type I) type I typically present with myoclonus, seizures, ataxia, cherry-red spot...
Development of a stable Sf9 insect cell line to produce VSV-G pseudotyped baculoviruses
Development of a stable Sf9 insect cell line to produce VSV-G pseudotyped baculoviruses
Baculoviruses have shown great potential as gene delivery vectors in mammals, although their effectiveness in transferring...
Preclinical dose response study shows NR2E3 can attenuate retinal degeneration in the retinitis pigmentosa mouse model RhoP23H+/−
Preclinical dose response study shows NR2E3 can attenuate retinal degeneration in the retinitis pigmentosa mouse model RhoP23H+/−
Retinitis pigmentosa (RP) is a heterogeneous disease and the main cause of vision loss within the group of inherited retin...
Correction: Preclinical safety and biodistribution of CRISPR targeting SIV in non-human primates
Correction: Preclinical safety and biodistribution of CRISPR targeting SIV in non-human primates
Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which...
Analysis and comparative evaluation of expedited programs for gene therapy products: insights from the United States, the European Union, Japan, and South Korea
Analysis and comparative evaluation of expedited programs for gene therapy products: insights from the United States, the European Union, Japan, and South Korea
Gene therapy products (GTPs) used for incurable diseases can be expedited for early commercialization to fulfill unmet nee...
AAV2 vector optimization for retinal ganglion cell-targeted delivery of therapeutic genes
AAV2 vector optimization for retinal ganglion cell-targeted delivery of therapeutic genes
Recombinant adeno-associated virus (AAV)-2 has significant potential as a delivery vehicle of therapeutic genes to retinal...
Retraction Note: Long noncoding RNA RBMS3-AS3 acts as a microRNA-4534 sponge to inhibit the progression of prostate cancer by upregulating VASH1
Authors and AffiliationsDepartment of Urology, The First Hospital of China Medical University, 110001, Shenyang, PR ChinaZ...
Correction: Cost of gene therapy
Correction to: Gene Therapy https://doi.org/10.1038/s41434-023-00408-y, published online 08 November ...
Ghrelin mediated cardioprotection using in vitro models of oxidative stress
Ghrelin mediated cardioprotection using in vitro models of oxidative stress
Ghrelin is commonly known as the ‘hunger hormone’ due to its role in stimulating food intake in humans. Howeve...
CRISPR/Cas9-mediated base editors and their prospects for mitochondrial genome engineering
CRISPR/Cas9-mediated base editors and their prospects for mitochondrial genome engineering
Base editors are a type of double-stranded break (DSB)-free gene editing technology that has opened up new possibilities f...
CSTB gene replacement improves neuroinflammation, neurodegeneration and ataxia in murine type 1 progressive myoclonus epilepsy
CSTB gene replacement improves neuroinflammation, neurodegeneration and ataxia in murine type 1 progressive myoclonus epilepsy
EPM1 is the most common form of Progressive Myoclonus Epilepsy characterized by late-childhood onset, ever-worsening and d...
Distributional comparison of different AAV vectors after unilateral cochlear administration
Distributional comparison of different AAV vectors after unilateral cochlear administration
The adeno-associated virus (AAV) gene therapy has been widely applied to mouse models for deafness. But, AAVs could transd...
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...
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