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Modulating Autophagy as a Treatment for Lysosomal Storage Diseases

Researchers at NIAMS have developed a technology for treatment of lysosomal storage diseases by inhibition of autophagy. Pompe disease is an example of a genetic lysosomal storage disease caused by a reduction or absence of acid alpha-glucosidase (GAA). Patients with Pompe disease have a lysosomal buildup of glycogen in cardiac and skeletal muscle...

Published: 8/14/2024 | Inventor(s): Cynthia Schreiner, Paul (Estate of) Plotz, Shoichi Takikita, Tao Xie, Rebecca Baum, Nina Raben

Keywords(s): Acid, A-glucosidase, Autophagy, B, Clearance, Complete, DEFICIENCY, Disabling, Disease, Enzyme, ERT, GB1XXX, GBXXXX, Genetic, GLYCOGEN, GXXXXX, IBXXXX, Inactivation, IXXXXX, Lysosomal, Model, Mouse, MUSCLE, Near, Patent Category - Biotechnology, Permits, Pompe, REPLACEMENT, SKELETAL, Storage, STORED, THERAPY, UA1XXX

Category(s): Collaboration Sought > Collaboration, Application > Therapeutics, TherapeuticArea > Rare / Neglected Diseases, Application > Diagnostics, Application > Research Materials

Method of Producing Immortalized Primary Human Keratinocytes for HPV Investigation, Testing of Therapeutics, and Skin Graft Generation

One of the major limitations of using cultured keratinocytes for research studies is that primary keratinocytes senesce after a few passages. Keratinocytes from specific anatomical sites are also difficult to culture. Scientists at the NIH have demonstrated that primary keratinocytes, from several anatomical sites, when treated with a small-molecule...

Published: 7/25/2024 | Inventor(s): Sandra Chapman, Jonathan Vogel, Atsushi Terunuma, Alison McBride

Keywords(s): AC4XXX, ACXXXX, AXXXXX, CC3XXX, CCXXXX, Complete, CXXXXX, DDXXXX, DXXXXX, EXTENDED, grafting, Human, IB2BXX, IB2XXX, IBXXXX, inhibitor, IXXXXX, KERATINOCYTES, Keratinocytes., Life-cycle, LONG-TERM, Maintenance, Papillomaviruses., Patent Category - Biotechnology, Persistence, Primary, PROLIFERATIVE, REPLICATION, ROCK, RXXXXX, SKIN, STATE, Studies., Sustain, Y-27632

Category(s): Collaboration Sought > Collaboration, Application > Diagnostics, Application > Therapeutics, Application > Research Materials, TherapeuticArea > Oncology, TherapeuticArea > Dermatology, TherapeuticArea > Infectious Disease

Attenuated Human Parainfluenza Virus (PIV) for Use as Live, Attenuated Vaccines and as Vector Vaccines

The identified technologies describe self-replicating infectious recombinant paramyxoviruses with one or more attenuating mutations, such as a separate variant polynucleotide encoding a P protein and a separate monocistronic polynucleotide encoding a V protein, or at least one temperature sensitive mutation and one non-temperature sensitive mutation....

Published: 7/25/2024 | Inventor(s): Peter Collins, Sheila Nolan, Mario Skiadopoulos, Brian Murphy

Keywords(s): cDNA, CIS-ACTING, Complete, DC5BXX, DC5XXX, DCXXXX, Determination, DXXXXX, FOR..., FRAMES, Gene, Generate, Generation, Genomic, HPIV2, HPIV2., HPIV2/V94, Identification, MEASLES, Mumps, MUTATION, Novel, Nucleotide, Overlapping, P/V, Parainfluenza virus type 3, Parainfluenza virus type 3; Human parainfluenza virus type 3, Reading, recombinant, rHPIV2, Separated, sequence, Strain, UA1XXX, USEFUL, V94

Category(s): Collaboration Sought > Collaboration, Collaboration Sought > Licensing, TherapeuticArea > Rare / Neglected Diseases, Application > Vaccines, TherapeuticArea > Infectious Disease