Amfani da kwayoyin halitta a matsayin magani: wannan shine ra'ayin da ke tattare da maganin kwayoyin halitta. Dabarun warkewa wanda ya ƙunshi gyaggyarawa kwayoyin halitta don warkar da cuta, maganin ƙwayoyin cuta har yanzu yana cikin ƙuruciya amma sakamakonsa na farko yana da alƙawarin.

Ma'anar maganin kwayoyin halitta

Maganin kwayoyin halitta ya ƙunshi gyare-gyaren kwayoyin halitta don rigakafi ko warkar da cututtuka. Ya dogara ne akan canja wurin kwayar halitta ta warkewa ko kwafin kwayar halitta mai aiki zuwa takamaiman sel, tare da manufar gyara lahani na kwayoyin halitta.

Babban ka'idodin maganin kwayoyin halitta

Kowane ɗan adam yana da kusan sel biliyan 70. Kowane tantanin halitta ya ƙunshi nau'i-nau'i 000 na chromosomes, waɗanda aka yi da filament mai siffar helix biyu, DNA (deoxyribonucleic acid). An raba DNA zuwa wasu 'yan dubunnan sassa, kwayoyin halitta, waɗanda muke ɗaukar kusan kwafi 23. Wadannan kwayoyin halitta sun hada da kwayoyin halitta, wani nau'in gado na musamman wanda iyaye biyu ke yadawa, wanda ya ƙunshi dukkanin bayanan da ake bukata don ci gaba da aiki na jiki. Lallai kwayoyin halitta suna nuna wa kowane tantanin halitta matsayinsa a cikin kwayoyin halitta.

Ana isar da wannan bayanin godiya ga lambar, haɗin musamman na tushen 4 na nitrogenous (adenin, thymine, cytosine da guanine) waɗanda ke yin DNA. Tare da lambar, DNA yana yin RNA, manzo wanda ya ƙunshi duk bayanan da ake buƙata (wanda ake kira exons) don samar da sunadaran, kowannensu zai taka muhimmiyar rawa a cikin jiki. Don haka muna samar da dubun dubatar sunadaran da ke da mahimmanci don aikin jikinmu.

Canje-canje a cikin jerin kwayoyin halitta don haka yana canza samar da furotin, wanda ba zai iya taka rawarsa daidai ba. Dangane da kwayar halittar da ta shafi, wannan na iya haifar da cututtuka iri-iri: ciwon daji, myopathy, cystic fibrosis, da dai sauransu.

Don haka ka'idar jiyya ita ce samar da, godiya ga kwayar warkewa, lambar daidaitaccen lamba domin sel su iya samar da rashin furotin. Wannan tsarin tsarin kwayoyin halitta da farko ya ƙunshi sanin ainihin hanyoyin cutar, kwayoyin halittar da ke tattare da su da kuma rawar da furotin ke yi.

Binciken ilimin halittar jini yana mai da hankali kan cututtuka da yawa:

• ciwon daji (65% na bincike na yanzu) 
• cututtuka na monoogenic, watau cututtuka waɗanda ke shafar kwayoyin halitta guda ɗaya kawai (hemophilia B, thalassemia) 
• cututtuka masu yaduwa (HIV) 
• cututtukan zuciya da na 
• cututtukan neurodegenerative (cututtukan Parkinson, cutar Alzheimer, adrenoleukodystrophy, cutar Sanfilippo)
• cututtuka na dermatological (cututtukan epidermolysis bullosa, dystrophic epidermolysis bullosa).
• cututtukan ido (glaucoma) 
• da dai sauransu.

Yawancin gwaje-gwajen har yanzu suna cikin binciken lokaci na I ko II, amma wasu sun riga sun haifar da tallan magunguna. Waɗannan sun haɗa da:

• Imlygic, maganin rigakafi na farko na oncolytic akan melanoma, wanda ya karɓi Izinin Kasuwancin sa (Izinin Kasuwanci) a cikin 2015. Yana amfani da kwayar cutar ta herpes simplex-1 da aka canza ta kwayoyin halitta don cutar da ƙwayoyin kansa.
• Strimvelis, na farko far dangane da kara Kwayoyin, samu ta Marketing izni a 2016. An yi nufi ga yara fama da alymphocytosis, wani rare kwayoyin rigakafi cuta ("kumfa baby" ciwo).
• Ana nuna miyagun ƙwayoyi Yescarta don maganin nau'in nau'i na nau'i biyu na m lymphoma ba Hodgkin: yada manyan B-cell lymphoma (LDGCB) da refractory ko relapsed primary mediastinal babban B-cell lymphoma (LMPGCB). Ya karɓi Izinin Tallanta a cikin 2018.

Akwai hanyoyi daban-daban a cikin ilimin halittar jini:

• maye gurbin kwayar halitta mai cuta, ta hanyar shigo da kwafin kwayar halitta mai aiki ko "jini na warkewa" a cikin kwayar da aka yi niyya. Ana iya yin wannan ko dai a cikin vivo: ana allurar kwayar cutar kai tsaye cikin jikin majiyyaci. Ko kuma in vitro: ana ɗaukar sel masu tushe daga kashin baya, an gyara su a cikin dakin gwaje-gwaje sannan a sake yin allura a cikin majiyyaci.
• gyare-gyaren genomic ya ƙunshi gyara maye gurbi kai tsaye a cikin tantanin halitta. Enzymes, da ake kira nucleases, za su yanke kwayar halitta a wurin da aka canza ta, sa'an nan kuma wani yanki na DNA ya ba da damar gyara kwayar halittar da aka canza. Koyaya, wannan hanyar har yanzu gwaji ce kawai.
• canza RNA, ta yadda tantanin halitta ya samar da furotin mai aiki.
• amfani da ƙwayoyin cuta da aka gyara, wanda ake kira oncolytics, don kashe ƙwayoyin cutar kansa.

Don shigar da kwayoyin halitta a cikin sel marasa lafiya, maganin kwayoyin halitta yana amfani da abin da ake kira vectors. Sau da yawa su ne ƙwayoyin cuta na ƙwayoyin cuta, wanda aka soke yuwuwar mai guba. Masu bincike a halin yanzu suna aiki akan haɓaka ƙwayoyin cuta marasa ƙwayoyin cuta.

A cikin 1950s ne, godiya ga mafi kyawun ilimin halittar ɗan adam, an haifi manufar maganin kwayoyin halitta. Koyaya, an ɗauki shekaru da yawa don samun sakamako na farko, wanda muke bin masu binciken Faransanci. A cikin 1999, Alain Fischer da tawagarsa a Inserm sun yi nasarar magance "kumburi na jarirai" da ke fama da matsanancin rashin ƙarfi na rigakafi da ke da alaƙa da X chromosome (DICS-X). Lallai kungiyar ta yi nasarar shigar da kwafin kwayar halittar da aka canza ta al'ada cikin jikin yara marasa lafiya, ta hanyar amfani da kwayar cutar kwayar cutar kwayar cutar retrovirus.