Controversial 3D. Probably none of you knows about Leber congenital amaurosis type 10, congenital blindness that affects 3 out of 100,000 children, condemning them to blindness for life. The only possible treatment is through gene therapy. 1/n
Thanks to Crispr-CAS9 technology we MODIFY the defective gene inside the patient eye ( they directly inject the treatment into the eye). Before CRISPR the treatment was based on Adenovirus carrying the correct form of the gene 2/n…
In both cases, we MODIFY the patient DNA and that's the only way (today) to treat this disease. LCA10 is not the only genetic disease affecting kids unfortunately the list is long and the variety of diseases is terrifying. 3/n…
Now think about a parent with a kid with a genetic disease, he/she would try and do anything to save him/her. ANYTHING and they would take some RISKS, gene therapy was a risk at the beginning, and any new experimental treatment shares a percentage of risk. 4/n
Now we come to #COVID19 and #vaccines. The new RNA vaccines are a HUGE scientific breakthrough, that may open new amazing therapeutic paths. And like ANY other human activity share a risk. 4/n…
FDA and EMA already cleared that risk to a very small percentage, probably lower than a normal drug. But there is a risk, and why should we take that risk? Because on the other hand, we have Covid19. A disease that killed 2 million people. 5/n
Covid19 can severely damage our body and it can cause long term damages (#LongCovid). 6/n…
People against the RNA vaccine (or vaccine in general) don't want to take a small risk ( FDA and EMA agree that is reasonably low) because they don't see the risk/reward ratio and you why? because they are SELFISH. 7/n…
Covid19 has a relatively low lethality that's why people are not scared, that's why people complain about social distancing, lockdown and wearing a mask because they are not directly affected. If it doesn't touch me, why should I be worried? that's so sad and miserable. 8/n
We need lockdown, social distancing, mask and vaccines to PROTECT the weak, the elderly, the immunocompromised. And why we do that? because we are human beings and we are better than wild animals.
So take a small risk to protect the others and stop complaining. 9/n
And maybe spend some times speaking with families hit by covid, speak to a person who has lost a parent, maybe you will realize that you are not the only one. We are one species and we help each other.
#WearADamnMask #vaccinoCovid 10/10
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More from @AurelianoStingi

13 Jan
3D polemico. Probabilmente nessuno di voi sa dell'amaurosi congenita di tipo 10 di Leber, cecità congenita che colpisce 3 bambini su 100.000, condannandoli alla cecità a vita. L'unico trattamento possibile è attraverso la terapia genica. 1 / n
Grazie alla tecnologia Crispr-CAS9 MODIFICHIAMO il gene difettoso all'interno dell'occhio del paziente (iniettano direttamente il trattamento nell'occhio). Prima di CRISPR il trattamento era basato su Adenovirus 2 / n…
In entrambi i casi MODIFICHIAMO il DNA del paziente e questo è l'unico modo (oggi) per curare questa malattia. La LCA10 non è l'unica malattia genetica che colpisce i bambini, purtroppo l'elenco è lungo e la varietà di malattie è terrificante. 3 / n…
Read 11 tweets
3 Jan
Ultimamente si è parlato molto della #varianteinglese e in generale delle possibi mutazioni che il #COVID19 può presentare. Ma perchè un virus muta? bhe le spiegazioni sono diverse ma semplificando al massimo 1/n
Il virus può mutare per due motivi:
1) intrinsencamente fa errori (non il caso del covid, muta molto meno di flu e HIV)
2) viene posta una 'pressione selettiva'
Per pressione selettiva intendo che noi proviamo in qualche modo ad ostacolarlo e lui fa di tutto per continuare a circolare ed infettare il più possibile. Nello schema ho riportato almeno due fattori che possono porre un pressione selettiva 4/n
Read 13 tweets
28 Dec 20
Ho notato che c'è parecchia confusione riguardo ai #vaccini a RNA come quello di #PfizerBioNTech. Il vaccino funziona grazie ad una molecola di RNA (istruzioni per costruire una proteina) viene inserita nella cellula (non nel nucleo) dove viene trasformata in una proteina 1/n
Non una proteina a caso ma Spike, la famosa chiave con quale #SARSCoV2 entra nelle nostre cellule. Una volta prodotta spike viene riconosciuta come estranea dal nostro sistema immunitario. Il vaccino esercita il suo ruolo, avverte ed addestra il nostro sistema immunitario. 2/n
Una volta che il nostro sistema immunitario incontra il vero virus intero è pronto a combatterlo e distruggerlo.
Read 9 tweets
14 Dec 20
Ci stiamo stressando (giustamente?) per gli eventuali effetti sconosciuti e a lungo termine di un vaccino, il 90% di noi manco sa la differenza tra DNA e RNA. Ma perchè non ci stressiamo altrettanto per gli effetti noti del #SARS_CoV_2. 1/n
La prof @VirusesImmunity ha pubblicato un paper dove mostra che Sars-CoV2 induce la produzione di auto-anticorpi che attaccano il nostro stesso organismo creando i danni seri e permanenti. 2/n
Inoltre con un mio collega abbiamo ipotizzato un meccanismo tumorigenico mediato da Sars-Cov2. Articolo ora in press. 3/n
Read 5 tweets
9 Dec 20
Good news from a therapy point of view in our fight against #SARSCoV2. A novel compound called EIDD-2801 blocks SARS-CoV-2 transmission in ferrets.
One of the compound that both reduce the symptoms and the TRANSIMISSION. 1/n
How does it work? This small drug is a ribonucleoside analogue, meaning a small fake letter of the virus alphabet. Providing this fake letter blocks the machinery that allows viral replication, resulting in virus death. 2/n
Researcher infected several ferrets with Sars-Cov2, later they put them with healthy ferrets (in the same cage) with or without this new drug. 3/n
Read 6 tweets
8 Dec 20
Update for DRUGS and TREATMENTS for #COVID19.
as today 8.12.20 only one drug is approved for covid19 treatment by FDA is the Remdesivir. 5 more treatments are EUA (emergency use approved) and me personally I added DEXA because of its efficacy. 1/n
Keep in mind that sometimes some drugs prove efficacy in trials but are not FDA approved for Covid treatment (and vice-versa). In the meantime more than 560 drugs are involved in trials at different stages. 2/n
Take home message: for the moment only one drug is approved to treat Covid19 but a lot of good candidates are on the pipelines, so there is hope!…………
Read 4 tweets

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