Rare & Genetic Diseases
| 9 min read
Rett syndrome currently has no cure. Now, innovations in gene therapy bring the possibility of treating the root cause of the disease closer than ever before.
Emerging CRISPR screening methods are shaping what’s possible in drug development and precision medicine.
| 9 min read
Drug repurposing trials for ichthyosis reveal complexity in the long-untreated disease.
| 4 min read
Previous genome editing techniques only work when delivered early in life. This approach could help preserve hearing that is lost later on.
| 7 min read
Achieving higher accuracy and depth in bottom-up proteomics involves navigating a multifaceted process with various steps and challenges.
| 3 min read
Epilepsy onset and seizure severity are linked to variants in the SCN2A gene, potentially leading to improved diagnostics and treatments.
| 5 min read
BioSpring and Pantherna agree on strategic cooperation in the field of genome editing
| 4 min read
A new method called “REVeRT” expands the capabilities of gene delivery platforms by transporting larger genes than previously possible.
| 5 min read
Biologics like monoclonal antibodies and mRNA vaccines are complex drugs. Yongchao Su uses biophysical tools and innovative strategies to understand them better.
| 5 min read
Researchers created an improved method for assays that detect viral vectors in a patient’s blood or urine. It’s used to ensure that bystanders will not be exposed.
| 10 min read
People with achromatopsia have never seen color. Restorative gene therapies have had mixed success, leaving researchers wondering why.
| 5 min read
The durability and capacity to boost protein expression of circular RNA makes it a promising candidate for next-generation vaccines and gene therapies.
Spatial transcriptomics technologies unveil a goldmine of biological information.
| 1 min read
Cutting-edge DNA synthesis technology propels scientific advancements and drug discoveries forward at unprecedented speeds.
| 3 min read
By watching brain organoids develop, researchers observed surprising abnormalities caused by mutations in a gene related to early-onset Alzheimer’s disease.
| 4 min read
A gene therapy targeting excitatory neurons significantly reduced seizures in a mouse model of the most common cause of child epilepsy.
Biobanking gives scientists access to thousands of biological samples, moving
precision medicine one step closer to reality.
| 8 min read
Alnylam Pharmaceuticals develops therapeutics based on the RNA interference (RNAi) pathway discovered in 1998.
| 3 min read
The pursuit of scientific knowledge relies on analytical thinking, but creativity also plays an essential role.
| 5 min read
Paolo Martini leads the biotech company’s rare disease research program with the hope of developing the very first treatments for genetic metabolic disorders.
Gene therapy has a complicated history, marked by many ups and downs and crafted by ever-advancing technologies.
| 10+ min read
Scientists identified specific genes that confer protection or vulnerability to various infections, pointing the way to new treatments.
| 6 min read
Experts discussed the major hurdles of AAV gene therapy, including immunogenicity, vector design, and AAV variability.
Page 1 of 19 - 361 Total Items
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A Technology Guide for CRISPR Screening
Emerging CRISPR screening methods are shaping what’s possible in drug development and precision medicine.
BioSpring and Pantherna agree on strategic cooperation in the field of genome editing
BioSpring and Pantherna agree on strategic cooperation in the field of genome editing
Charting a cellular treasure map with spatial transcriptomics
Spatial transcriptomics technologies unveil a goldmine of biological information.
