I ask my friend Hottod and all users for help.for a few months now, I've been trying to see beyond what is officially on the pitch. the territory on which my couriousness is undermined opens up to a scenario that completely upsets both the value of 2591 and the potential value that we must expect on the market. to be clear, if a multiple of 4 is now plausible, the evaluation moves to multiples of multiples of multiples.I am very convinced of what I propose personally, but this is my problem.In recent months there have been initiatives that also affect us closely due to the relationship we have with Acadia, which cannot leave us indifferent.
https://ir.acadia-pharm.com/news-re...armaceuticals-and-stoke-therapeutics-announce
https://www.pharmaceutical-technology.com/news/roche-shape-gene-therapy-deal/
https://www.geekwire.com/2021/seatt...ises-112m-develop-rna-editing-gene-therapies/
https://investors.beamtx.com/node/7711
https://locanabio.com/press-release...enerative-neuromuscular-and-retinal-diseases/
our friends and associates of Patent 2591
https://www.amo-pharma.com/news/Press_Release_211222.htm
reasonably, I think the PW trial we are designing is the turning point. there are substantial elements that consolidate this thesis which, if confirmed, binds us to a revolutionary scientific course on which Neuren is light years ahead in consideration of the fact that he is the holder of highly confidential information acquired during the Rett study shared with Acadia, with the difference that 2591 has distant origins and was also the subject of preclinical study by the American army. we have a database of families involved in studies that others do not have and which must be acquired with years of experimentation.
https://www.genecards.org/cgi-bin/carddisp.pl?gene=PWRN2
https://www.futuremedicine.com/doi/pdf/10.2217/epi.15.55
Prader-Willi syndrome is a neurodevelopmental disorder that is characterized by infantile hypotonia, feeding difficulties, hypogonadism, mental deficiency, hyperphagia (leading to obesity in early childhood), learning problems, and behavioral difficulties. A paternal 15q11-q13 deletion is found in ∼70% of patients with Prader-Willi syndrome, ∼25% have uniparental maternal disomy 15, and the remaining 2% to 5% have imprinting defects. The proximal deletion breakpoint in the 15q11-q13 region occurs at 1 of 2 sites located within either of 2 large duplicons allowing for the identification of 2 deletion subgroups. The larger, type I (TI) deletion involves breakpoint 1, which is close to the centromere, whereas the smaller, type II (TII) deletion involves breakpoint 2, located ∼500 kilobases distal to breakpoint 1. Breakpoint 3 is located at the distal end of the 15q11-q13 region and common to both typical deletion subgroups. Analyses of the genetic subtypes of Prader-Willi syndrome to date have primarily compared individuals with typical deletion and uniparental maternal disomy 15 without grouping the individuals with a deletion into TI or TII. Distinct differences have been reported between individuals with Prader-Willi syndrome resulting from deletion compared with uniparental maternal disomy 15 in physical, cognitive, and behavioral parameters. We previously presented the first assessment of clinical differences in individuals with Prader-Willi syndrome categorized as having type I or II deletions. Adaptive behavior, obsessive-compulsive behaviors, reading, math, and visual-motor integration assessments were generally poorer in individuals with Prader-Willi syndrome and the TI deletion compared with subjects with Prader-Willi syndrome with the TII deletion or uniparental maternal disomy 15. Four genes (NIPA1, NIPA2, CYFIP1, and GCP5) have been identified in the chromosomal region between breakpoints 1 and 2 and are implicated in compulsive behavior and lower intellectual ability observed in individuals with Prader-Willi syndrome with TI versus TII deletions. We quantified messenger-RNA levels of these 4 genes in actively growing lymphoblastoid cells derived from 8 subjects with Prader-Willi syndrome with the TI deletion (4 males, 4 females; mean: age 25.2 ± 8.9 years) and 9 with the TII deletion (3 males, 6 females; mean age: 19.5 ± 5.8 years). Messenger-RNA levels were correlated with validated psychological and behavioral scales administered by trained psychologists blinded to genotype status. Messenger RNA from NIPA1, NIPA2, CYFIP1, and GCP5 was reduced but detectable in the subjects with Prader-Willi syndrome with the TI deletion, supporting biallelic expression. For the most part, messenger-RNA values were positively correlated with assessment parameters, indicating a direct relationship between messenger-RNA levels and better assessment scores, with the highest correlation for NIPA2. The coefficient of determination indicated the quantity of messenger RNA of the 4 genes explained from 24% to 99% of the variation of the behavioral and academic parameters measured. By comparison, the coefficient of determination for deletion type alone explained 5% to 50% of the variation in the assessed parameters. Understanding the influence of gene expression on behavioral and cognitive characteristics in humans is in the early stage of research development. Additional research is needed to identify the function of these genes and their interaction with gene networks to clarify the potential role they play in central nervous system development and function.
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