Isifo se-Gaucher

Isifo se-Gaucher yisifo esiyingqayizivele esiyingqayizivele, okuholela ekubutheni kwezinye izitho ezithile (ngokuyinhloko esibindi, isibungu kanye nomnkantsha wamathambo) wama-deposit ethize. Isikhathi sokuqala lesi sifo sabonakala futhi sichazwa udokotela waseFrance uPhilip Gaucher ngo-1882. Wathola amangqamuzana athile ezigulini ezine-pleen ekhulisiwe, lapho amafutha angaziwa khona. Kamuva, lawo maseli aqala ukubizwa ngokuthi ama-Gaucher cells, futhi lesi sifo, ngokulandelana, isifo se-Gaucher.

Izifo zokugcina izilonda

Izifo ezingenayo i-lysosomal (izifo zokuqoqa lipids) yigama elivamile lezifo eziningi eziphilayo ezihambisana nokuphazanyiswa kwe-intracellular cleavage yezinto ezithile. Ngenxa yokungaziphathi kahle nokuntuleka kwamakhemikhali athile, izinhlobo ezithile ze-lipids (isibonelo, i-glycogen, i-glycosaminoglycans) azihlukanisi futhi azigcini emzimbeni, kodwa zibuthelwa kumaseli.

Izifo ze-lysosomal azivamile. Ngakho-ke, ejwayelekile kunazo zonke - isifo se-Gaucher, senziwa ngesilinganiso semvamisa ye-1: 40000. Imvamisa inikezwa ngokwesilinganiso ngoba lesi sifo sinesifayela ngokwezinga lokuziqhenya ngokweqile futhi kwezinye izinhlanga ezivaliwe zingase zivele izikhathi ezingaphezu kuka-30 kaningi.

Ukuhlukaniswa kwezifo ze-Gaucher

Lesi sifo sibangelwa isici esakhiweni esithinta ukuhlanganiswa kwe-beta-glucocerebrosidase, i-enzyme evuselela ukuhlanza kwamafutha athile (glucocerebrosides). Kubantu abanalesi sifo, i-enzyme edingekayo ayanele, ngoba amafutha awahlukanisi, kodwa ahlangane kumaseli.

Kunezinhlobo ezintathu zezifo ze-Gaucher:

  1. Uhlobo lokuqala. Ifomu eliphansi kunazo zonke futhi elivela njalo. Ekhishwa ukunyuka okungenakuphuza kwesiteji, ukwanda okuncane kwesibindi. Isistimu enkulu yezinzwa ayithintekile.
  2. Uhlobo lwesibili. Ifomu elivela njalo ngenomonakalo omkhulu we-neuronal. Kuzibonakalisa ngokuvamile ezinsana ezisencane futhi ngokuvamile kuvame ukuholela ekufeni.
  3. Uhlobo lwesithathu. Ifomu le-juvenile subacute. Ijwayele ukutholakala ngokuvamile eneminyaka engu-2 ukuya kwengu-4. Kunezilonda zesimiso se-hematopoietic (i-bone lomnkantsha) kanye nesilonda esingenalutho se-system yezinzwa.

Izimpawu Zesifo Sezinkukhu

Lapho lesi sifo, amaseli e-Gaucher aqoqa kancane kancane ezithombeni. Okokuqala kukhona ukwanda okungenakuqhathaniswa esitezini, bese isibindi, kunezinhlungu emathanjeni. Ngokuhamba kwesikhathi, ukuthuthukiswa kwe- anemia , thrombocytopenia, ukuphuma kwegazi ngokuzenzekelayo kungenzeka. Uhlobo lwe-2 no-3 lwesifo, ubuchopho kanye nesistimu yezinzwa njengento yonke iyathinteka. Ngohlobo lwe-3, esinye sezibonakaliso eziphawuleka kakhulu zomonakalo ohlelweni lwezinzwa kungukuphulwa kokunyakaza kwamehlo.

Ukuxilongwa kwezifo ze-Gaucher

Isifo se-Gaucher singatholwa ukuhlaziywa kwamangqamuzana weglucocerebrosidase gene. Kodwa-ke, le ndlela yinkimbinkimbi kakhulu futhi iyabiza kakhulu, ngakho-ke isetshenziselwa izimo ezingavamile, lapho kutholakala kunzima ukuxilongwa kwesifo. Ngokuvamile, ukuxilongwa kuyenziwa lapho amangqamuzana e-Gaucher etholakala ematheni okwephulwa noma ubhedu obukhulu ngesikhathi se-biopsy. Imifanekiso yamathambo ingasetshenziselwa ukukhomba ukuphazamiseka kwezici ezihlobene nomonakalo wamarrow.

Ukwelashwa kwezifo ze-Gaucher

Kuze kube manje, indlela kuphela ephumelelayo yokwelapha lesi sifo - indlela yokwelapha okufaka indawo nge-gluccerase, isidakamizwa esithatha indawo ye-enzyme elahlekile emzimbeni. Kusiza ukunciphisa noma ukunciphisa imiphumela yomonakalo womzimba, ubuyisele imetabolism evamile. Ukuhambisa izidakamizwa Kudingeka ukuthi kusetshenziswe njalo, kepha ku-1 no-3 uhlobo lwesifo bayasebenza ngempela. Ngefomu elibi lesi sifo (uhlobo lwesi-2) kuphela ukwelashwa kwesondlo kuyasetshenziswa. Futhi, ngezilonda ezinzima zezitho zangaphakathi, ukususwa kwe-spleen , ukuguqulwa kwamathambo okusawoti kungenziwa.

Ukutshala izihlahla zamathambo noma ama-cell stem kubhekisela ekwelashweni okunamandla kakhulu ngokulingana nokufa kwabantu futhi kusetshenziselwa kuphela ithuba lokugcina uma ezinye izindlela zokwelapha zingasebenzi.