Ke hoʻohana nei i nā genes ma ke ʻano he lāʻau lapaʻau: ʻo ia ka manaʻo ma hope o ka lāʻau lapaʻau. ʻO ka hoʻolālā lāʻau lapaʻau e pili ana i ka hoʻololi ʻana i nā genes e hoʻōla i kahi maʻi, aia nō ka lāʻau lapaʻau i kona wā kamaliʻi akā ke hoʻohiki nei nā hopena mua.

Ka wehewehe ʻana o ka lāʻau gene

Hoʻopili ʻia ka hoʻōla ʻana o ke ʻano genetically e pale a hoʻōla i ka maʻi. Hoʻokumu ʻia ia ma ka hoʻoili ʻana o kahi gene therapeutic a i ʻole kope o kahi gene hana i loko o nā cell kikoʻī, me ka manaʻo e hoʻoponopono i kahi kīnā genetic.

ʻO nā kumu nui o ka lāʻau lapaʻau

ʻO kēlā me kēia kanaka he 70 billion mau pūnaewele. Loaʻa i kēlā me kēia keena he 000 mau chromosomes, i hana ʻia me kahi filament ʻano helix pālua, DNA (deoxyribonucleic acid). Hoʻokaʻawale ʻia ʻo DNA i nā ʻāpana liʻiliʻi, nā genes, kahi a mākou e lawe ai ma kahi o 23 kope. Hoʻokumu kēia mau genes i ka genome, kahi hoʻoilina hoʻoilina kū hoʻokahi i hoʻoili ʻia e nā mākua ʻelua, aia nā ʻike āpau e pono ai no ka hoʻomohala ʻana a me ka hana ʻana o ke kino. Hōʻike maoli nā genes i kēlā me kēia cell i kona kuleana i loko o ke kino.

Hāʻawi ʻia kēia ʻike ma muli o kahi code, kahi hui kūʻokoʻa o nā kumu nitrogenous 4 (adenine, thymine, cytosine a me guanine) i hana i ka DNA. Me ke code, hana ʻo DNA i ka RNA, ka ʻelele i loaʻa nā ʻike āpau e pono ai (kapa ʻia nā exons) e hana ai i nā protein, e pāʻani kēlā me kēia mea i kahi kuleana kūikawā i ke kino. No laila, hana mākou i nā ʻumi tausani o nā protein e pono ai no ka hana o ko mākou kino.

ʻO ka hoʻololi ʻana i ke kaʻina o kahi gene no laila e hoʻololi i ka hana ʻana o ka protein, ʻaʻole hiki ke hoʻokani pololei i kāna kuleana. Ma muli o ke ʻano e pili ana, hiki i kēia ke alakaʻi i nā ʻano maʻi like ʻole: nā maʻi maʻi, myopathies, cystic fibrosis, etc.

No laila, ʻo ke kumu o ka lāʻau lapaʻau e hāʻawi, mahalo i kahi gene therapeutic, kahi code kūpono i hiki i nā cell ke hana i ka protein nele. ʻO kēia kaʻina hana mua e pili ana i ka ʻike pono ʻana i nā ʻano hana o ka maʻi, ke ʻano i pili a me ke kuleana o ka protein e hoʻopaʻa inoa ai.

ʻO ka noiʻi ʻana o Gene therapy e pili ana i nā maʻi he nui:

• nā maʻi kanesa (65% o ka noiʻi o kēia manawa) 
• nā maʻi monogenic, ʻo ia hoʻi nā maʻi e pili ana i hoʻokahi gene (hemophilia B, thalassemia) 
• nā maʻi lele (HIV) 
• cardiovascular mai 
• nā maʻi neurodegenerative (maʻi o Parkinson, maʻi Alzheimer, adrenoleukodystrophy, maʻi Sanfilippo)
• nā maʻi dermatological (junctional epidermolysis bullosa, dystrophic epidermolysis bullosa)
• nā maʻi maka (glaucoma) 
• etc.

Aia ka hapa nui o nā ho'āʻo ma ka noiʻi ʻana I a i ʻole II, akā ua hopena kekahi i ke kūʻai ʻana i nā lāʻau lapaʻau. Aia kēia mau mea:

• ʻO Imlygic, ka immunotherapy mua oncolytic e kū'ē i ka melanoma, i loaʻa iā ia ka Mana Kūʻai Kūʻai (Marketing Authorization) i ka makahiki 2015. Hoʻohana ia i kahi maʻi herpes simplex-1 i hoʻololi ʻia e hoʻopili ai i nā maʻi kanesa.
• ʻO Strimvelis, ka lāʻau lapaʻau mua e pili ana i nā cell stem, loaʻa i kāna Mana Kūʻai Kūʻai ma 2016. Ua manaʻo ʻia no nā keiki e loaʻa ana i ka alymphocytosis, kahi maʻi kūlohelohe kūlohelohe ("bubble baby" syndrome)
• ua kuhikuhi ʻia ka lāʻau ʻo Yescarta no ka mālama ʻana i ʻelua ʻano lymphoma non-Hodgkin aggressive: diffuse large B-cell lymphoma (LDGCB) a refractory a i ʻole relapsed primary mediastinal large B-cell lymphoma (LMPGCB). Ua loaʻa iā ia kāna Mana Hoʻolaha ma 2018.

Aia nā ala like ʻole i ka gene therapy:

• ka hoʻololi ʻana i kahi gene maʻi, ma ka lawe ʻana i ke kope o kahi gene hana a i ʻole "genera therapeutic" i loko o kahi cell target. Hiki ke hana i kēia ma ka vivo: ua hoʻokomo pololei ʻia ka gene therapeutic i loko o ke kino o ka mea maʻi. A i ʻole in vitro: lawe ʻia nā pūnaewele mai ka spinal cord, hoʻololi ʻia i loko o ka hale hana a hoʻihoʻi hou ʻia i loko o ka mea maʻi.
• ʻO ka hoʻoponopono genomic ka hoʻoponopono pololei ʻana i kahi hoʻololi genetic i loko o ke kelepona. ʻO nā enzyme, i kapa ʻia ʻo nucleases, e ʻoki i ke ʻano ma ke kahua o kona hoʻololi ʻana, a laila hiki i kahi ʻāpana DNA ke hiki ke hoʻoponopono i ka gene i hoʻololi ʻia. Eia naʻe, he hoʻokolohua wale nō kēia ala.
• hoʻololi i ka RNA, i mea e hoʻopuka ai ke kelepona i kahi protein hana.
• ka hoʻohana ʻana i nā maʻi maʻi i hoʻololi ʻia, i kapa ʻia ʻo oncolytics, e pepehi i nā maʻi maʻi maʻi.

No ka loaʻa ʻana o ka gene therapeutic i loko o nā keʻena o ka mea maʻi, hoʻohana ka lāʻau lapaʻau i nā mea i kapa ʻia he vectors. He mau vector viral pinepine lākou, ua hoʻopau ʻia ka mea ʻona. Ke hana nei nā mea noiʻi i ka hoʻomohala ʻana i nā vectors non-viral.

ʻO ia i ka makahiki 1950, mahalo i ka ʻike ʻoi aku ka maikaʻi o ka genome kanaka, ua hānau ʻia ka manaʻo o ka lāʻau lapaʻau. Eia nō naʻe, he mau ʻumi mau makahiki ka loaʻa ʻana o nā hopena mua, a mākou i ʻaiʻē i nā mea noiʻi Farani. I ka makahiki 1999, ua mālama ʻo Alain Fischer a me kāna hui ma Inserm i ka mālama ʻana i "nā pēpē pēpē" i loaʻa i kahi immunodeficiency hui pū ʻia i hoʻopili ʻia me ka X chromosome (DICS-X). Ua lanakila maoli ka hui i ka hoʻokomo ʻana i kahi kope maʻamau o ka gene i hoʻololi ʻia i loko o ke kino o nā keiki maʻi, me ka hoʻohana ʻana i ka retrovirus-type viral vector.