Search
Search Results
-
Mesenchymal Stem Cell-Mediated Suicide Gene Therapy
An ocean of evidence points toward feasibility of applying gene therapy-based methods to manifest a prolonged remedial impact. Mesenchymal Stem Cells...
-
TCR Gene Therapy for Cancer
The protocol describes the procedure of antigen-specific T cellT-cell generation and TCR T-cell receptor (TCR) identification for the use in...
-
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. The shock-and-kill cure...
-
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 genetic disorders of the brain....
-
Gene Therapy in Liver Disease: Challenges and Outcomes
Liver-directed gene therapy (LDGT) has been proposed as a means of treating many single-gene inherited disorders. LDGT has been used to create...
-
Vector Shedding and Immunogenicity Sampling in AAV-Based Gene Therapy for Retinitis Pigmentosa Patients
Viral vectors are employed in gene therapy to deliver functional genes. It is often traceable in bodily secretions and excretions of the recipients....
-
AAV gene therapy for hereditary spastic paraplegia type 50: a phase 1 trial in a single patient
There are more than 10,000 individual rare diseases and most are without therapy. Personalized genetic therapy represents one promising approach for...
-
Intravitreal Delivery of PEGylated-ECO Plasmid DNA Nanoparticles for Gene Therapy of Stargardt Disease
ObjectiveCurrent gene therapy of inherited retinal diseases is achieved mainly by subretinal injection, which is invasive with severe adverse...
-
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 spots, and blindness because of...
-
Gene therapy for inborn errors of immunity: past, present and future
Inborn errors of immunity (IEI) are diseases caused by genetic mutations that affect the immune system’s ability to fight pathogens, cope with the...
-
Unveiling a novel fusion gene enhances CAR T cell therapy for solid tumors
The efficacy of Adoptive Cell Transfer Therapy (ACT) in combating hematological tumors has been well-documented, yet its application to solid tumors...
-
Evaluating Gene Therapy as a Potential Paradigm Shift in Treating Severe Hemophilia
Hemophilia is characterized by a deficiency in coagulation factors VIII or IX. The general standard of care for severe hemophilia is frequent...
-
Combinatorial gene therapy for epilepsy: Gene sequence positioning and AAV serotype influence expression and inhibitory effect on seizures
Gene therapy with AAV vectors carrying genes for neuropeptide Y and its receptor Y2 has been shown to inhibit seizures in multiple animal models of...
-
Clonal selection of hematopoietic stem cells after gene therapy for sickle cell disease
Gene therapy (GT) provides a potentially curative treatment option for patients with sickle cell disease (SCD); however, the occurrence of myeloid...
-
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. However, potential concerns...
-
Molecular and Biochemical Assessment of Gene Therapy in the Canine Model of Duchenne Muscular Dystrophy
Mutations in the dystrophin gene result in Duchenne muscular dystrophy (DMD), a progressive muscle-wasting disease. Adeno-associated virus (AAV)...
-
CRISPR-Cas and CRISPR-based screening system for precise gene editing and targeted cancer therapy
Target cancer therapy has been developed for clinical cancer treatment based on the discovery of CRISPR (clustered regularly interspaced short...
-
Recent Advances on Therapeutic Approaches for Friedreich’s Ataxia: New Pharmacological Targets, Protein, and Gene Therapy
Friedreich ataxia (FA) is an inherited autosomal recessive neurodegenerative disorder. The most common mutation in FA is caused by a (GAA)n triplet...
-
-
Preexisting Neutralizing Antibodies against Different Adeno-Associated Virus Serotypes in Humans and Large Animal Models for Gene Therapy
Gene therapy is a potential cure for several inherited retinal dystrophies, and adeno-associated virus (AAV) has emerged as a vector of choice for...