Repeated dosing of AAV-mediated liver gene therapy in juvenile rat and mouse models of Crigler-Najjar syndrome type I.

Crigler-Najjar syndrome is an ultra-rare monogenic recessive liver disease caused by gene mutations. Complete UGT1A1 deficiency results in severe unconjugated hyperbilirubinemia in newborns that, if not treated, may lead to brain damage and death. Treatment is based on intensive phototherapy, but its efficacy decreases with age, rendering liver transplantation the only curative option.

Posterior Lateral Meniscal Root Repair Through Lateral Tunnel and Anterior Cruciate Ligament Revision: How to Avoid Tunnel Overlapping.

Posterior lateral meniscal root (PLMR) tears are commonly observed in conjunction with anterior cruciate ligament (ACL) injuries. The presence of PLMR tears exacerbates knee instability, increasing stress on ACL grafts and accelerating joint degeneration if left untreated. Anatomical repair of PLMR tears is therefore crucial for restoring native knee kinematics and reducing tibiofemoral contact pressures, thereby safeguarding the ACL graft.

Characterizing Leishmania infantum-induced resistance to trivalent stibogluconate (SbIII) through deep proteomics.

Leishmania infantum belongs to the L. donovani complex, which includes species associated with visceral leishmaniasis. Traditionally, antimonial compounds have served as the primary antiparasitic treatment for all clinical forms of leishmaniasis.

Deciphering Chemical Rules for Drug Penetration into Strongyloides.

Strongyloidiasis, a parasitic infection, presents a significant public health challenge in tropical regions due to the limited repertoire of effective treatments. The screening of chemical libraries against the therapeutically relevant third-stage larvae (L3) of the model parasite yielded meager success rates. This situation is reminiscent of Gram-negative bacteria, where drug entry is a limiting factor.