The treated patient is a baby who has a few pains or a severe combined immunodeficiency disorder (SCID). Door ena DNA-fout in de bloedstamcellen maken SCID-patiënten geen afweercellen aan en kan het lichaam zich niet verdedigen tegen infections. Immediate stem cell therapy can correct DNA damage. “The stunned stamps are in the form of a patient who has been able to develop a good waiting system,” said Arjan Lankester, a high school pediatrician at Stamceltransplantatie. “The baby is dealing with problems without having to react to a disorderly treatment of vaccinations. A fantastic result.
Cruyway
Drie maanden na de geboorte werden er bij de pationt stamcellen afgenomen. In the DNA of this cell, a good version of the hood was made, and the RAG1 gene was found to be depleted. “Here we have a virus,” said Frank Staal, Hoogleraar Stamcelbiologie, uit. “We have discovered that the virus is the source of the virus in its genome in the DNA of its host, but it is believed that all other sources. The virus is the only one of the crucian carts that die on the right side. Vervolgens werde derepareerde cellen teruggegeven aan de pationt, die binnen een maand he ziekenhuis mocht verlaten. Standing in Lancaster, we are waiting for the patient with this complex, but it is a unique treatment for the living genesis to be.
This is your own donor
Without handling overdue children with SCID vaak voor hun eerste levensjaar. Maar aan de huidige behandeling, namelijk stamceltrantplantatie, kleven een aantal nadelen. So it is not possible for a good donor to be able to provide a transplant with the help of another person who is at risk of risk. “All patients with stampentherapy are of their own, we have these problems,” said Lankester. Werder onderzoek moet uitwijzen of deze therapie in de toekomst de nieuwe standaard wordt for SCID.
Prijskaartje
De stamcelgentherapie wordt gemaakt in a special laboratory. The LUMC has one of the most important stores in the Netherlands, and the creation of genetically modified cellular products has taken place. “The fact that we are not in the pharmaceutical industry,” said Staal. Dat neemt niet weg dat deze therapie, net als andere gentherapieën, erg kostbaar is. “The therapy of the therapy is now in the underworld, but the patient is not. Op de lange termijn is de hoogte van het prijskaartje nog onduidelijk ». The price of a subsidiary is highly reasonable. “It takes a lot of time to get a job out of a group of people who can be treated with therapies.” Bijvoorbeeld met richtlijnen voor beleidsmakers nad de vergoeding ».
