BiovaxID, a personalized therapeutic vaccine against B-cell lymphomas [0.03%]
BiovaxID：一种针对B细胞淋巴瘤的个性化治疗性疫苗
Milan Reinis
Milan Reinis
Biovest International Inc (a subsidiary of Accentia BioPharmaceuticals Inc), under license from Stanford University, is developing BiovaxID, a personalized therapeutic vaccine against B-cell lymphomas that, in combination with GM-CSF, exclu...
Diamyd, an alum-formulated recombinant human GAD65 for the prevention of autoimmune diabetes [0.03%]
一种用于预防自身免疫性糖尿病的重组人GAD65药物Diamyd（含明矾）
Simon A Hinke
Simon A Hinke
Diamyd Medical AB is developing Diamyd (GAD-65), an alum formulation of a full-length recombinant human glutamic acid decarboxylase 65 for subcutaneous injection, for the potential prevention and treatment of type 1 diabetes (T1DM) or laten...
NT-501: an ophthalmic implant of polymer-encapsulated ciliary neurotrophic factor-producing cells [0.03%]
NT-501：包封了睫状神经营养因子产生细胞的聚合物眼用植入体
Dwaine F Emerich,Christopher G Thanos
Dwaine F Emerich
Neurotech Pharmaceuticals Inc is developing NT-501, an implantable polymeric device containing a genetically modified cell line that secretes ciliary neurotrophic factor, for the potential treatment of retinitis pigmentosa (RP) and age-rela...
Olga Mykhaylyk,Olivier Zelphati,Joseph Rosenecker et al.
Olga Mykhaylyk et al.
Magnetofection is defined as the magnetically enhanced delivery of nucleic acids associated with magnetic nanoparticles and has been utilized to deliver synthetic siRNAs to cultured cells. Certain magnetic nanomaterials associate with siRNA...
Sigrid Nikol
Sigrid Nikol
Coronary and peripheral artery diseases are highly prevalent and characterized by an unmet requirement for medical treatment in the more advanced stages. These shortcomings comprise the requirement for vessel regeneration using therapeutic ...
Hematopoietic stem cell gene therapy in Hurler syndrome, globoid cell leukodystrophy, metachromatic leukodystrophy and X-adrenoleukodystrophy [0.03%]
造血干细胞基因治疗在亨特综合征、格洛布茨细胞白质营养不良、元氨酸白质营养不良和X-肾上腺白质营养不良中的应用
Nathalie Cartier,Patrick Aubourg
Nathalie Cartier
Hurler syndrome, metachromatic leukodystrophy, globoid-cell leukodystrophy (Krabbe's disease) and X-linked adrenoleukodystrophy are inherited diseases of the CNS that can be cured or arrested by allogeneic hematopoietic stem-cell transplant...
Vanessa Gabrovsky,Michele P Calos
Vanessa Gabrovsky
Gene therapy for hemophilia A has fallen short of success despite several clinical trials conducted over the past decade. Challenges to its success include vector immunogenicity, insufficient transgene expression levels of Factor VIII, and ...
Shannon M Conley,Xue Cai,Muna I Naash
Shannon M Conley
For multiple reasons, the eye is an excellent organ for gene therapy and while nonviral gene therapy modalities have been known for quite some time, they have only been applied to the eye in the past decade. Despite significant advances in ...
Rahul Srinivasan,David J Fink,Joseph C Glorioso
Rahul Srinivasan
Chronic pain is a serious debilitating condition affecting a large number of individuals and with substantial societal impact in terms of quality-of-life and absence from work. Current therapies often exhibit activity at sites other than th...
Interactions of adenovirus vectors with blood: implications for intravascular gene therapy applications [0.03%]
腺病毒载体与血液的相互作用：应用于血管内基因治疗的意义
Alan L Parker,Stuart A Nicklin,Andrew H Baker
Alan L Parker
Despite various obstacles the promise of gene therapy has begun to be realized, as demonstrated by the successful phenotypic correction of X-linked SCID in infants. Although ex vivo gene therapy is advantageous, many diseases, for example, ...